Board of Directors a Dynamic Component of Society Check out the

Transcription

Volume 35 | Number 1 | Spring 2011
COURAGE
Board of Directors
a Dynamic
Component of
Society
Check out the 2010
recap and welcome
your new board
members
Annual Fund
Raises $85,591
Critical funding
stream keeps our
organization strong
Walk/Run
Program
Celebrates 11th
Anniversary
Nationwide events
raised nearly
$3.1 million since
inception
Upcoming
Events
2011 family
conference to be
held July 28–30
in St. Louis
Stop and
Refuel Through
Therapy
An inside
perspective on
how it can
help YOU
Do you have a personal story
or an article idea for a future
issue of Courage? Please
write to us and remember
to send photos!
MISSION STATEMENT
The National MPS Society exists to find cures
for MPS and related diseases. We provide hope
and support for affected individuals and their
families through research, advocacy and awareness
of these devastating diseases.
The National MPS Society’s office (ground floor on left).
Submission
Cutoff Date
Issue
Jan. 1.......................Spring
April 1....................Summer
July 1.............................Fall
Oct. 1....................... Winter
To submit information to Courage, please send text
(preferably via e-mail) to the address at right. Photos
should be labeled whenever possible. Please note cutoff
dates. Any information received after these dates will
be included in the subsequent issue.
The articles in this newsletter are for informational
purposes only, and do not necessarily reflect the opinions
of the National MPS Society and its board of directors.
We do not endorse any of the medications, treatments or
products reported in this newsletter, and strongly advise
that you check any drugs or treatments mentioned with
your physician.
Courage reserves the right to edit
content as necessary.
National MPS Society
PO Box 14686
Durham, NC 27709-4686
t: 877.MPS.1001
p: 919.806.0101
f: 919.806.2055
E-mail:
[email protected]
www.mpssociety.org
Table of
CONTENTS
Membership &
Subscription Form
President’s Letter.............................................................................. 2
Executive Director’s Letter................................................................ 3
Name
Development Director’s Letter........................................................... 4
Program Director’s Letter.................................................................. 4
Financial Summary........................................................................... 5
Affected Individual’s Name
Date of Birth
New Members.................................................................................. 6
Donations......................................................................................6–9
2010 Recap..............................................................................10–11
Upcoming Events........................................................................... 11
Diagnosis
Relationship
Address
New Board Members................................................................12–13
Medical Travel Assistance Program................................................. 13
OSCAR Awards.............................................................................. 14
Making Headlines........................................................................... 15
City, State, ZIP
Telephone
E-mail
Annual Fund..............................................................................16–21
Family News..............................................................................22–23
Holiday Photos..........................................................................24–25
Fundraising................................................................................26–31
Standing Ovation.......................................................................32–33
A Warm Welcome........................................................................... 34
Remembering Our Children............................................................ 35
Family
Foreign
Professionals
b $50.00
b $80.00
b $75.00
Corporate Memberships Available
Would you like your name to appear
in our online directory? b YES b NO
Would you like to receive Courage, the
Society’s newsletter? b YES b NO
Legislative Update.....................................................................36–37
Research News: Hope for the Future.........................................38–41
Resources | Helpful Information.................................................42–51
MPS Classifications........................................................................ 52
Board of Directors.......................................................................... 53
Pictured on the cover:
Susan Quade (MPS IV)
Lucas Montgomery (MPS III) and mom Stacey
Livia Hubert (MPS III)
Would you like our publications in
b electronic (e-mailed) format or
b hardcopy (mailed) format
Please send your membership form and
check to:
PO Box 14686
Durham, NC 27709-4686
1
President’s Letter
It is my distinct honor, pleasure and
privilege to return to the role of
president of this fine organization.
Though I left the post only four short
years ago, it seems like it has been an
eternity. It seems that way due to the
speed of change occurring in the MPS
community. Both within the Society
and without, progress is being made by
leaps and bounds.
As for curing our kids, we’ve never been closer!
There is approved enzyme replacement therapy
IV drugs for MPS I, II and VI, and a clinical trial
has started for MPS IV A. Clinical trials also
are under way to treat the brain’s intrathecally
for MPS I and II, and a clinical trial is in the
beginning stages for MPS III A. Dog studies
have proven MPS I is virtually nonexistent when
treating dogs from birth with IV and intrathecal
enzyme replacement therapy, and newborn
screening will soon be a reality for some of the
MPS diseases in certain states. While progress
for some of the syndromes has lagged behind
others, we will not rest until therapies are
available for all.
While the therapies I’ve mentioned are not cures,
they are huge steps in the right direction while
other therapies or cures are discovered—and
there are many under consideration: from gene
replacement therapy to chaperone therapy to
substrate deprivation therapy and many others.
It is very exciting and much different than it was
in 1994 when my children were diagnosed.
As for supporting our families, we’ve never
done more! In addition to the wonderful
educational family conferences each year, the
Society distributes the quarterly magazine
Courage, and produces booklets and fact sheets
with educational information on our syndromes
2
and other important topics. We provide direct
family financial assistance through dues waivers,
continuing education scholarships, durable
medical goods, conference travel scholarships,
Extraordinary Experiences, and our newest
program—medical travel assistance (see page
44). In addition, we continue to advocate for
you, our families, in Washington, DC, through
sponsoring legislation, securing much needed
research dollars, and obtaining access to quality
healthcare.
This kind of progress can only be made by the
coordinated efforts of many parties. First, I must
thank all of you who support this organization
with your time and financial resources and
respond to our pleas for assistance when needed.
Second, I thank your hard-charging board of
directors whose dedication to the cause never
ceases to amaze me (find out more information
on our newest board members on page 12).
Third, I thank our devoted and capable staff
that carries out the mission of the Society on a
daily basis which allows us as parents to see to the
needs of our families. Lastly, I thank our many
partners in the MPS community—our physicians,
researchers, therapists and pharmaceutical
friends. Only with the concerted efforts of all
of these parties can we provide the support and
hope that all of our children deserve!
I wrote a lot of thank you notes the first months,
then life became busy as I assumed responsibility
for our newsletter, the family conference,
and the increasing interactions with the
pharmaceutical companies as the MPS I phase
III enzyme replacement therapy (ERT) clinical
trial progressed and TKT (now Shire) was
gearing up for the MPS II ERT phase I/II clinical
trial. So I hired Laurie Turner and she wrote the
thank you notes. Then Laurie became busy as
she began working closely with families and our
family support programs, because I was building
bridges with our MPS family foundations and
U.S. lysosomal disease advocacy groups, plus
interacting with the international MPS advocacy
groups. Along the way, we enlarged our research
grants program, added an online donor database,
hired a development director, added new family
support programs, improved our Web site and
increased communications with our members.
So Laurie hired someone to help her write the
thank you notes.
the ERT for MPS I. The FDA calls an Advisory
Committee hearing if additional information is
needed before approval can be given for a new
medicine. This was the first ERT for an MPS
disease. The implications were huge. We asked
families to speak at the hearing and worked
with them on their presentations. They were
magnificent. The tension in the room that day
was palpable, gradually breaking as, one by one,
the committee members voiced their “yes” to
approve Aldurazyme. This set the stage for all
future FDA approvals of ERT for MPS diseases
and was a very emotional day for all involved.
Along the way I have worked with amazing people.
Our board members can never be recognized
enough for their gifts of time, dedication and
expertise. They are the driving force of the
Society; the staff works on their behalf. I admire
and respect each of them.
What does the future hold? I believe we’re on the
cusp of major changes. After years of treatment
possibilities being tested in labs around the
world, these treatments are breaking through
translational research into clinical trials. We
must maintain the momentum and continue to
support the work of these dedicated researchers.
It’s an honor working for this great organization.
Oh, and I do still write the occasional thank you
note!
Executive Director’s Letter
Ten years ago I was hired as the Society’s executive
director. The first executive director stayed only
six months; he did not want to talk to families. At
the time, I was serving on the board of directors,
so I had a good understanding of the work of the
Society. When we were discussing my potential
hire, the then president, Linda Shine, said the
majority of my work would be writing thank you
notes to donors. I thought, “I can do that. My
mama taught me how to write thank you notes.”
Well, you get the picture. In 10 years the Society
has grown—a lot—with the programs we offer,
and we’ve added staff and an office that is not
in a volunteer parent’s spare bedroom. There
are many adjectives to describe those 10 years:
challenging, inspiring, exciting, sad and even
overwhelming at times. Some of our children
are living longer, more satisfying lives while we
continue to look to the future for treatments
for others. Every call to the office about another
child passing creates tears but pushes us to
work harder for that ultimate goal of a cure for
everyone with MPS.
Someone asked what was my most significant
moment. Without a doubt, it was the 2003 FDA
Advisory Committee hearing for Aldurazyme®,
3
Development | Program Directors’ Letters
4
Spring is in the air, and reflecting
on 2010 accomplishments, the
National MPS Society says thank
you! Our fundraising and walk/
run programs raised more than
$400,000 for research and family
assistance. In 2010, the Sponsor
a Child for a Cure program raised
more than $10,000! Whether you
hosted a large or small event, each
one made a difference and we are honored you
continue to work on behalf of our families to
raise awareness and funds.
walk/run program that funds general research.
We also cannot forget to say thank you for the
fundraising events that hosted walk/runs for
syndrome-specific research. Also in this issue is
information on the new Friends and Neighbors
program (FAN).
We are always looking for creative ideas for
fundraising efforts. If you have any suggestions
or are interested in serving on the Fundraising
Committee, please contact me directly at terri@
mpssociety.org. We look forward to a successful
2011 fundraising year.
Inside this issue of Courage are program highlights
from the successful 2010 Annual Fund and
Spring is always a busy time for
the Family Support Committee,
and this year will be no different.
They have been working on two
of the toughest assignments this
committee faces: processing the
conference scholarships and the
Continuing Education Scholarship
(CED) applications. In reading the
CED essays, it is common for the
tears to flow—both in laughter and in sadness—
over the shared feelings and emotions in the
written words. The essays that are submitted are
some of the most powerful stories ever read, and
with the common MPS bond, all of the committee
members know the place deep within your heart
and soul where the love for your MPS family
member fills your life and helped these amazing
essays come to paper. It is heartwarming to read
the stories of siblings, and the love and special
bonds shared. The love of a parent going back
to school to honor their child, and the amazing
essays written by individuals who are battling the
challenges of MPS daily. Thank you for submitting
your words, your love, your stories, and allowing
us to read them. Thank you committee members
for all you do to make the programs a continued
success.
The Family Support Committee also has been
busy helping to create a new program—the
Medical Travel Assistance Program (MTAP). See
page 13 to see if your family will be able to apply
for a grant to assist in transportation costs for
medical appointments more than 200 miles from
your home.
I hope to see you in St. Louis at our 25th Annual
Family Conference!
The Society annually obtains an audit of its financial activity and files a Form 990 with the IRS.
For additional information, contact the Society’s office.
2010 Actual*2011 Budget
Beginning Cash Balances
Restricted for Endowment, Research
$ 1,737,859
$ 1,837,076
and Family Assistance
Unrestricted
612,040 1,007,799
Inflows
Annual Fund
Conference Income
Donations/Fundraisers — General
Donations/Fundraisers — Family Assistance
Donations/Fundraisers — Research
Dues
Interest/Investment Gain/(Loss)
Operating Grant/Sponsored Revenue
Corp. Gifts/Foundation Grants/Major Gifts/Endowment
Walk/Run Research Fundraiser
Sale Items
Total Inflows
$ 1,708,175
Outflows
Administrative
$
Bereavement
Conference
Education — Newsletters, Booklets, Web Page, Video
Family Assistance — Direct
Fundraising
Legislative
Membership Database and Directory
Office and Equipment
Personnel
Research
Sponsored Expenses
Total Outflows
85,262
136,328
120,345
67,345
175,065
33,830
50,534
398,000
380,000
252,058
9,408
$ 1,097,500
43,202
$
6,862
156,218
54,846
32,672
72,593
5,986
19,987
45,246
220,450
457,500
97,636
$ 1,213,199
90,000
60,000
125,000
68,000
161,500
35,000
45,000
170,000
90,000
245,000
8,000
Financial Summary
National MPS Society Financial Report Summary
51,000
5,700
177,000
202,800
69,200
62,000
10,000
18,500
53,000
241,032
400,500
365,000
$ 1,655,732
Ending Cash Balances
$ 1,837,076
$ 1,590,045
Restricted for Endowment, Research and Family Assistance
Unrestricted 1,007,799
696,599
* 2010 Unaudited data
5
New Members
Ross Berning
Verona, WI, father of Colin Berning, MPS III A
Jennifer and John Besse
Lincoln, MO, parents of Mikal Besse, MPS III A
Crystal Cervantes
San Bernardino, CA, sister of David Cervantes,
MPS II
Melanie DeFilippis
Old Bridge, NJ, parents of Mark DeFilippis, ML III
Karen Fish
Homer, IL, grandmother of Livia Grace Hubert,
MPS III A
Elizabeth and Chris Hoff
Balaton, MN, parents of Willow and Bella Hoff,
MPS IV
Jennifer Hutcheson
Broadway, VA, mother of Carlton Hutcheson,
MPS I
Donations
In Honor of
Cecilia Bien
Cheryl Bien*
Lorraine and Frank Bien*
Matt and Joan Houser
Rachel Adams
Kevyn and Stacey Adams
Michael Bodura Jr.
Helen Fallon*
William and Alayne
Lowenberger*
Sam Aker
Dean and Kathy Aker*
Conner Anhalt
Mel and Millie Anhalt*
Roy and Janine Levitch
Mel and Millie Anhalt
Caroline Masur
Millie Anhalt
Charles and Ellyn Phillips*
Millie Anhalt’s birthday
Mike and Babs Eaker
Stephanie Annis
Tim and Mikki Annis*
Davis Barkley
Amy Barkley*
Dwight and Debbie Barkley*
Anna Funkhouser*
Robert Harvey and
Roxanne Maffitt*
Andrew Kramer*
6
Jayden and Brooklyn Boyce
Tara Ann Blazer*
Earline Dickerson*
Richard and Mary Losee*
Minnie Wood*
Annabelle Bozarth
William J. Anderson Jr.
Justin Calixto
Kimberly Cook
Kien Do
Candace Duncan
Ragan Hughs
Patsy Lindsey
Micol Merriman
Kristine Morris
Russell and Alexander
Browne
Mary Ann Messick*
Nikki Schiebel
Steve and Tina Sharpe
Matthew Caldwell
Mark and Stephanie
Caldwell
Lori Ledoux
Karen Larson
Balaton, MN, grandmother of Willow and Bella
Hoff, MPS IV
Cooky M. Mays
Houston, TX, grandmother of Annabelle
Bozarth, MPS IV
Anthony Murphy
Daly City, CA, adult with MPS I
Cassandra Reamer
North Branch, MI, mother of Austin William
Reamer, MPS III B
Margaret Spinale
Medford, MA, friend of Joey Raftery, MPS III B
Michelle and Alan Vitulich
Westport, CT, aunt and uncle of Austin Noll,
MPS III C
Ron and Kim Wiest
Chesterfield, VA, parents of Noah Wiest, MPS VI
Aidan Carter
Bill and Susan Johansen
Cindy and Ricky Fabian
Lou Fabian*
Sam Caswell
Denise Caswell*
Heidi and Keith Caswell*
Robinson Funeral
Home, Inc.
Braden Farrell
Betty and Barney Arceneaux*
Bruce and Tasha Farrell
T.J. Catanzarite
Pamelia Farthing*
Steve and Bryce Chesser
Kenneth and Barbara
Velten*
Bryce Chesser
Dr. Don Westerland*
Jim Zambrano
Mitchell Finzel
David and Stacey Aronson*
Donald and Ramona Finzel*
Nic and Susan McPhee
Gilbert*
Kimberly Fowler
Donald and Mary Jane Fowler
Jeffrey and Barbara Harrell*
Jack Frye
Kevin and Tracey Litwin*
Mackenzie Grace Clark
Barbara Ives
Sabrina Gabriel
Roland and Linda Tamayo
Amanda Crawford
George and Linda Johnson
Marcus Garvin
Margaret Offutt*
Denise Dengel
Darlene Sweeney
Taylor Geary
Gary and Karen Brugnoli
Sharon Glasscock*
Allison Deutser
Cynthia and Randy Anhalt
Sue Deutser’s birthday
Cayden Duncan who is
helping his friend
Annabelle Bozarth
Ina Miller
Bryant B. Graeber
Blake and Ashley Graeber*
Karina Guajardo
Jerry and Charlotte Campbell
Ross and Belia Coddington*
Charles and Josephine
Ellard*
continued
Jake Hampton
Wallis, Monica, Natalie and
Nicole Hampton*
Lyle, Jade and Rylie Hays
Jerry and Elizabeth Highfill
Rylie Hays
Larry and Barbara Hays*
Anita Hodgkins
Macy’s Corporate Services, Inc.
Ricky Hodgkins
Mike and Carolyn Managan*
Rhonda Pare
Kris and Brian Klenke
Clarence and Faye Brown*
Mrs. Ellen Krech’s birthday
Trey Lane
Steve Cuomo*
Estee Leger
Carol Morrison
Kay Levy’s birthday
Olivia Lovell
Robert and Marjorie Lovell*
Mary and John Hoffman
Helen and Bill Vespse*
Allison and Lacey Lukondi
Susan Lukondi
Madison and Laynie Holland
James and Margaret Myrick
Jack Mahony
David and Ada Alden*
Stephen Baum and
Brenda Baker*
Ewa Robinson*
Dominic Smith
Michelle Hopkins
John and Janet Arendt*
Livia Hubert
Jessie DeHaan*
Ryan and Braydon Kapes
Beth Valocchi*
Allison Kirch
Bob and Eve Bonville*
Barb Fisher*
Herbert Kirch*
Larry and Susan Kirch*
Tom Kirch*
Ann O’Malley*
Jenny Klein
Terri Klein*
Branden Patterson
Ralph and Jane Daniels*
Erin Peters
Sergio and Angela Stasi*
Ellen Phillips’ birthday
Logan Piefer
Sally Armstrong*
Darrell and Kay DiStefano*
Lewis Whitaker Jr.*
John Thornton
Michael and Susan Thornton
Julie Thorsrud
Hal and Laura Thorsrud
Shannon Tootill
Janet Rafferty*
Zachary Townsend
Leonard and Phylis
Greenstein*
Michael “Tyler” Trahan
Phyllis Wilson
Shihjay Wang
Rex and Sharon Wang*
Maria Ratto
John and Debby Ratto
John and Mary Ratto*
Tina Ratto
Matt Wells
Claude and Roselyn Wells*
Donald and Lisa Wells*
Wilbur and Irene Erhardt*
Gary Markowitz’s birthday
Evan Reed
Floyd, Sharon and
Kerry Reed*
Scotty Whitecotton
Lennie Forkas*
Rita Haan
Kayla Martin
B.D. Ashe*
Nathan Roma
Jonathan and Lisa Dols*
Mason Whittle
Pamela and Darrell Whittle*
Dylan Michael McClurg
Stephen and Marie McClurg*
Luke Sarantinos
A.L. and Angela Sarantinos*
Aleis Wilson
Victor and Pamela Kovach*
Morgan and Blake McDermott
Leo and Janet Cook*
Mrs. Joyce Schechter’s
birthday
Zayd Jawad
Muhanad Hawaad and
Jumana Shawkat*
Amy and Murray Kalmin’s
birthdays
Carlo A. Otero
Carlos Otero*
Becky and Patrick Thomas’
wedding
Marcia Hass
Thomas Shinu
Chip and David Radius
Janet Krosschell
Logan Marcotte
David and Regina Marcotte*
Wyatt and Gavin Jones
Molly Wilhelmson
Jeffrey Orr
John Williams*
Tracy Szemanski
Gail Finney
Katelyn Vanghle
Barb and Mike Weikel*
Allan Iezzi in honor of
his grandchildren Oliver
and Waverly
Mack Trucks, Inc.
Dorian and Wynn Johnson
Lorena Cummings
Elizabeth Harlow
Pauline Hernandez
Kaitlyn and Sadie O’Donnell
Fred and Ronda O’Donnell*
Jeff Stribing
Lancaster Lions Club*
Chad Pyper
Deborah Toga*
Glen Malina
Barbara and George Malina*
Sydnee Jensen
Loretta Shaw*
Austin F. Noll IV
Austin and Dorothy Noll*
Lawrence Pung*
Sarah Mallia
Joseph and Jeanne Mallia
Sunni Markowitz
Kallie Alyssa McGuire
Diane McGuire Cooper*
Loy and Susan McKeithan
Harold and Charlene
McKeithen
Danny Miller
Sandra Babel*
William and Peggy Cook*
Brian Shingle*
John and Diane Stabb*
Austin T. Nace
Jacqueline A. Fenaroli*
Tyler Seabrook
Nancy Bucher
Sasha Segal’s birthday
Intellivest Financial
Services, LLC
Michael Sheridan
Robert Sheridan*
Trent Silkey
David and Rebecca Silkey
Donations
In Memory of
Paul Adams
Mary Starr Adams*
Stuart and Karin Adams
Robert Brulhardt Jr.*
Raimund and Inge Kasner
Frances McCauley*
continued
Lara Smith
Fred Smith
7
Kristopher Arnold
William and Margaret
Hughes*
Raymond and Julie O’Hara*
Erich Nikolas and
Garrett Curtis Baise
Wade V. Baise*
Erma T. Getchell
Central Assembly of God
Gabriela Giannone
Cathryn Coppola*
Jo Ann Scarpelli
David, Randy and
Jared Barrett
Samuel and Donna Petersen
and family*
Danny Gniazdowski
Gary and Debbie Ball*
David and Leesa Galloway*
Tom and Anne Gniazdowski*
Robert and Constance
Martineau*
Freeman Bauerle
Ruth Bauerle*
Kevin Golke
Diana Golke
Tommy Bennett
Julia Bradley
Cameron Green
Maggie Been
Craig and Kelli Clark
Lindsey Clarke-Lusk
Kevin and Sheila George
Debbi Gomez
Paul and Laurie Howard
Kerry Hunsaker
Howard Kelfer*
Kristine Klenke
Seniz Meyers
Penny Thomas
Emily Vowels
Katy and Jay Weible
Tom and Kim Whitecotton
Mark Bergquist
Donald and Nina Bergquist*
Mark Bernbaum
Karas family*
Joan Werner*
Ray Bryant
Randall Kane
Edie Burke
Harold and Charlene
McKeithen
Ward McKeithen*
Greg Sharrits*
Pat, Cristie and Cody Taylor
Joshua Burt
Robert Steiner
Timothy Carter
Nancy Kearney
Bradley Chapman
Kenneth and Victoria Warner*
Devon Clark
Saundra and Larry Clark*
Robert Steiner
Karlee Rae Classen
Kent and Karlene Classen*
Greg and Scott Day
Julia Smith*
David Downing
Family of David Downing*
Jamie Drummer
Kathleen Jones*
Sean Dummann
Carl Dummann
Katie Foshay
Maria and Steven Foshay*
Rishi Garg
Harry and Janice
Van Horne*
8
Maggie and Keegan Hackett
John and Cheryl Hackett*
Garner Halvorsea
Jim and June Murphy
Grant Hickman
Todd and Lorna Albert
Bob and Sue Maher
Rob and Annette Musrck
Shelby Middle School staff
David and Jeanne Tarvin
Bonnie Lynn Hoegl
John and Bonnie Hoegl
Elizabeth Hogan
Joan Giammarino
Spencer Holland
Donna Brinkley*
Amanda Keith
Ralph and Martha Anderson*
Barbara Russo*
Andrew and Louise
Zygmuntowicz*
Kraig Klenke
Stuart and Karin Adams
Pamela Alford
Valerie and Steve Barnett
Steve and Alice Bartels
Patricia Beard
Beth Abert Anchor Home
Health Care
Diane Boda
Wilbur and Esther Boda
Bollwerk, Ryan and
Tartlow, LLC
Nancy Brada
Kenneth and Joann Brinker
Dennis and Peggy
Brueggemann
Bruce and Karma Colbert
Stephen and Denise Clayton
Mike and Michelle Dapkus
Ernie and Debbie Dummann
Douglas and Lori Dunbar
Michael and Kathryn Eller
Jamie and Jason Fowler
Moe and Diana Frey
Steve and Cindy Gnaedinger
Dorothy K. Grange, MD
Kenneth and Linda
Grotefendt
Hamel Seed and Farm
Supply, Inc.
Richard and Donna
Hellinger
Kenneth and Sheryl Hencke
Cory and Sheryl Hess
Dave and Linda Hooks
Paul and Laurie Howard
Randall Hunt
Nancy Kearney
Mr. and Mrs. Edwin Klenke
Hilbert and Verna Klenke
Martha Klenke
Randy and Cheryl Klenke
Robert and Ruth Klenke
Rodney and Valerie Klenke
Roger and Lori Klenke
Richard and Joyce Koenig
Robert and Janet Kruse
Vanessa Laughlin
Glenn Leduc
Dorothy and Nelson
Linenfelser
Denis and Linda Litteken
Gene and Barb Lotter
Steve and Monica Martin
Mathew and Joanna McCrea
Cindy Miller
Beth Patrick
Amy Petty
Leslie Phillips
Daryl and Tammy Riechmann
Leo and Darlene Rode
Rosa Schultz
Jeffrey and Hortensia Smith
Robert Steiner
Randy and Sandy Strohmeier
James and Betty Voss
Lee and Janet Voss
Richard and Karla Warren
Doris Watson
Tracy and April Watson
Gladys Wilkening
Gordon Wingate
K.K. Wopat
Terry and Jeannette Zobrist
Terry and Cindy Zweig
Maggie and Joey Langford
James and Carolyn Stultz*
Joseph Burke and
Lauren Langlin
J.E. and M.D. Woodruff
Mark K. Lessing Jr.
Mr. and Mrs. Frank Arena
BMG Enterprises, LTD
Karen Bliss
Susan and Dennis Burke
David Carlino
David and Maureen
Cunningham
James and Susan Davison
Rita Denigris
Thomas Dillion
Christopher and
Ann Donaldson
Michael and Arlene Flanagan
Gail Tuthill AFI Foodservice
Richard Frost
Melchor Garcia
Andrew and Angela Hamm
Christopher and Cathleen
Hunter
Stephen and Kathleen Hurst
Alana Katz
Martin and Louise Kennedy
Carl and Maria Lehnert
Mary Loesch
Peggy Mahoney
Brenna McCarthy Merced
Phillip and Marita Mulholland
Laura Pelegrino
Barbara and Keith Pushee
Daniel and Donna Rinaldi
Robert Schipf Babylon
Village Youth Lacrosse
James and Julia Rogers
Albert Scerbo
Bonnie Schappert
Nancy Sforza
Anthony and Susanne
Sgueglia
The Cleveland Foundation
Wendy Tigchelaar
Joseph and Alice Vanderbelt
Maureen Walsh
Colleen West-Levy
Logan LoCascio
Lloyd and Cheryl LoCascio*
continued
Alyssa Louden
Donald and Karen Louden
Anthony M’Sadoques
Rene and Patricia
M’Sadoques*
Jenny Wingate
Wayne Howser*
George and Athena
Sarantinos*
Tara Matthews
Fay Matthews*
Michael Zechender
Kathryn Bullard*
Glenn Miller
Glenn and Kathy Miller*
Joe Burke and Marilyn
Patricia Langin
Mitch Moore
Cherylynne Moore
Matching
Gifts
Cade Morrissey
Ron and Chris Morrissey*
Chris Myers
Mr. and Mrs. W. Frank
Rogers Jr.*
Daniel Nardin
Bernadine Nardin
Nancy Notley
David and Frances Notley*
Adam Rattman
Sue Rattman*
Diana Rodrigues
Isabella Rodrigues*
Daniel Rudny
Michelle Towell
Dianne Saitta
Michael and Marcia Geller
Mihail Sawyer
Nancy Kearney
Phyllis Schroeder
General Kinematics
Peggy Shipley
Mountain Home Baptist
Church
Ronald and Judy Nelson
LeRoy and Marge Shipley
Jack Straub
Jim and June Murphy
Zachary Szemanski
Gail Finney
Emma Turner
Krista Baker*
Peter Wehrle
Bernard Ray*
Don Westerland
Steven and Karole Chesser
Richard White III
Richard and Margaret
White*
Abbott Laboratories, Inc.
Employee Giving Campaign
Automatic Data Processing, Inc.
Bank of America
Matching Gifts*
Emerson Network Power
Fidelity Investments IBM
Retiree Charitable Campaign
Hewlett-Packard
IBM Employee Charitable
Contribution Campaign
Johnson & Johnson Matching
Gifts Program
Pfizer Foundation Matching
Gifts Program
PG&E Corporation Campaign
for the Community
Leo Burnett USA
McGraw-Hill
Fundraisers
Daniel’s garage sale
hosted by Mike and Traci
Bosch in honor of Daniel
“Squeek” Bosch
Arbonne fundraiser
hosted by Dawn Boster in
honor of Rebekah Lehto
Cornhole fundraiser
hosted Rob and Lisa Muller
in honor of Riley Muller
Klenke Bowl-a-thon –
10th Anniversary
hosted by Brian and Kris
Klenke in memory of
Kraig Klenke
Cabi clothing party
hosted by Lynn Hopkins in
honor of Michelle Hopkins
Sowden Walk for a Cure
hosted by Joshua and
Sheri Sowden
Johnson Halloween fundraiser
hosted by Chris and
Mercedes Johnson in honor
of Dorian and Wynn
Hendrick harvest fundraiser
hosted by Cynthia Anderson
in memory of her son
David Hendrick
Rylie’s Runners for Research
hosted by Lyle and Jade
Hays in honor of Rylie Hays
Forest Park bake-off contest
hosted by Frank Monge in
honor of Will Luthcke
Cayden’s birthday fundraiser
hosted by the family of
Cayden Duncan in honor
of Annabelle Bozarth
Holiday Annual Fund
for Taylor
hosted by Sharon Glasscock
in honor of Taylor Geary
Curry wedding fundraiser
hosted by Ryan and
Kelly Curry
Logan Piefer Walk-a-thon
hosted by Kathy Greenberg’s
Basic Life Skills class in
honor of Logan Piefer
Donations
Bob Ahern*
Christa Armstrong
Sherri and Scott Baggett*
Leonard and Barbara
Barcousky*
Jane Barker *
Shawn Barkley*
Pat Barrett Jr.
Marie Bonville*
Jerry and Charlotte Campbell
Larry and Margaret Cohen*
Rocco Colicchia
Brooke and Stephen Cornwell*
Bob and Laura Cowin*
Henry and Barbara DeVries
Patricia DiLuzio
Marjorie Ditmore*
Jeffrey and Mary Dozbaba*
Ernie and Debbie Dummann*
John Exner*
Janet Forde
Stephen and Betsy Fowler*
Eileen Gideon*
Michael and Frances Goldberg*
Mark Haskins*
Arthur and Brenda Hawkins*
Anita Hodgkins
Linda Horn*
John and Yvette Iannelli
James and Mary Kalteux*
Heidi Kanealy*
Kathleen Kimura*
Laura Kinto
Janet and Gary Kirch*
Larry and Margaret Kirch
Cohen*
James and Norvella Koelling*
Laurel Creek Hardwoods, Inc.*
Lynn Lilore*
David and Kiersten Lowe
Eileen Marchica*
Richard and Janice Marino*
James and Eleanor McCaffrey*
James and Kimberly
McClelland
Pauline and Robert Michek*
Gary Mortensen*
Soraida Munoz*
Elizabeth Neufeld*
Anne Novak*
Norma and Lawrence Pacl
Steven Page
Thomas and Vickie Patterson
Dave and Michelle Patterson*
Personal Assistant Services
of Colorado
William Peterson
Douglas and Mary Pica*
Katy and Jay Plotkin*
Crystal Polis*
Sam and Nancy Ramsey
John and Debby Ratto
George and Carol Rice*
Mr. and Mrs. A. Kay Rogers*
Jo Ann Scapelli
Ron Shebik*
Mike and Barbara Smith
William and Meghan Stark*
Robert and Susan Steiner
Jeffrey and Kimberly Stribing*
Steven and Lynn Szydel*
Becky Tanmachi*
Jaime Tighe
John Timmons
Nita Todd*
Sarah Aaserude and
Todd Waddell
Stephen and Martha Waters*
Gary and Tracy Wells*
James and Elaine Wiesman
* Annual Fund donor
9
2010 Recap
2010 Board of Directors
The bylaws of the National MPS Society require that the board of directors meets
three times each year. Following is a general summary from those meetings; the
complete meeting minutes can be obtained by calling the Society’s office.
The first board meeting of 2010 was held in Miami, FL, on Feb. 13. Dr. Stephen
Walkley, chair of our Scientific Advisory Board, presented an overview of the scientific presentations
from the World conference, held that week in Miami.
• Discussed new projects for 2010: updating the Web site, membership survey and tri-fold floor
displays
• Discussed addition and logistics of CYCLE and adult conferences to this year’s family conference
• Discussed 40 percent increase in family fundraisers since 2008
• Discussed changes in 2009 donations (14 percent decrease in walk/runs, 6 percent increase in
Annual Fund), projections for 2010, grants to be submitted and new fundraisers
• Approved 2010 budget and research grants to award
• Approved the walk/run policy and the T-shirt policy
• Approved revisions to the conflict of interest policy
• Approved committee assignments for 2010
• Approved $5,000 increase in family support budget
• Approved Governance Committee as a standing committee (bylaw change)
The second board meeting was held July 17, 2010, in Seattle, WA.
• Discussed launch of International MPS Network Web site in April (impsn.org)
• Discussed new clinical trials, MPS IV and MPS III A
• Presented Web site and Facebook demographics and statistics
• Announced Denise Dengel’s acceptance to serve as chairperson of the 2010 Annual Fund
• Discussed development strategies to apply only for grants $25,000 or more and develop planned
giving program while increasing networking with the community
• Discussed increased advertising of the Sponsor a Child for a Cure program, allowing all families to
participate in walk/runs
• Announced new fact sheets being developed: Developmental Testing, IEP tips, Endocrine, newly
diagnosed families interested in UCBT; Guardianship fact sheet completed
• Discussed the 2010 family support programs awarded to date: 20 education and conference (17
family, one cycle and 10 adult) scholarships, nine FAP grants, four Extraordinary Experiences,
and two regional social events
• Discussed May legislative trip to DC including NIH and SSA meetings, NORD dinner • Austin Noll presented his PowerPoint FDA testimony, with a focus on biomarker endpoints and
acceptable study designs
• Reviewed and approved the 2011 officer ballot. The following officers were elected for two-year
terms:
– President: Steve Holland
– Vice President: Kimberly Frye
– Treasurer: Tom Gniazdowski
– Secretary: Austin Noll
• Approved bylaw changes
• Approved 2010 grants awarded, approved renewing the 2009 grants for the second year
• Approved moving money from security investments following the treasurer’s report
10
continued
2010 Recap
The final board meeting of the year was held prior to the 24th Annual Family
Meeting in Buena Park, CA.
• Reviewed details of the three conferences and associated events being held and responsibilities
of board members
• Discussed soliciting members through Survey Monkey to obtain national list of therapists and
physicians
• Reported that 31 families will participate in the SACFAC program
• Announced that the “Fundraising Road to a Cure” display will launch at the conference
• Presented overview of member survey which will be presented at the AGM and in Courage
• Discussed the need to budget for conference scholarships for families diagnosed after
scholarships have been awarded and increasing attendance of newly diagnosed families
• Discussed allowing greater leniency for the GPA on the CED application
• Reviewed the Web site difficulties with the walk/run registrations and efforts being made to rectify
• Said goodbye to retiring board members Ernie and Debbie Dummann, Klane and Amy White,
Tami Slawson
• Discussed the travel policy; majority voted against amending
• Approved removing cap of $200 for FAP application
• Approved the board election ballot presented by the Governance Committee
• Approved Boston for the 2012 family conference
• Approved San Antonio for the 2013 family conference
Upcoming Events
National MPS Society 2011 Family Conference
The 25th Annual National MPS Society Family Conference will be held July 28–30, 2011, in
St. Louis, MO. The beautiful Sheraton Westport Chalet Hotel is nestled in St. Louis’ impressive West
Port Plaza area, just minutes from some of the city’s most popular attractions. West Port Plaza offers
access to more than 30 restaurants, exclusive shops and entertainment. Save the date to attend this
conference that promises to offer all the updates on research, medical care and topics that you’ve
requested.
Registration materials are available on our Web site. The deadline for early bird registration is
May 1, 2011.
11
New Board Members
Welcome New Board Members!
In 2010 we sadly said goodbye to Ernie and Debbie Dummann, Tami Slawson, and Klane and
Amy White, all of whom chose not to run for another term on the Society’s board of directors.
As a testament to the commitment of these dedicated and hardworking board members, each has
requested to remain active by serving on board committees. We are very grateful to receive their
ongoing gifts of time, plus their knowledge and expertise.
On Jan. 1, 2011, we welcomed our new board members: Stephanie Bozarth, Hope and Dave Madsen,
and Jeff Bardsley.
The National MPS Society welcomes new board members (l. to r.) Jeff Bardsley, Dave Madsen,
Hope Madsen and Stephanie Bozarth.
Stephanie and her husband Austin husband live
in Alexandria, VA. They have two daughters,
Annabelle, age 4, who was diagnosed with
MPS IV A at age 6 months, and Madeline, age 2.
Stephanie has a master’s degree in social work
and is a licensed clinical social worker with
psychotherapist experience in areas of individual
and family counseling, grief, bereavement,
medical diagnosis adjustment, parenting,
crisis intervention, depression and behavior
management. She has hosted the charming
Mad Hatter Tea fundraiser, in addition to other
creative fundraisers.
“I want to make a significant difference for
my daughter and all families affected by MPS
and related diseases,” said Stephanie. “When
Annabelle was diagnosed, my husband and I
had never heard of mucopolysaccharidoses. It
was an overwhelming and difficult time during
the early diagnosis phase. I want to expand my
contribution beyond fundraising activities to
further increase awareness, scientific research,
peer support and community support to
ultimately improve the lives of those with MPS
and related diseases.”
12
Stephanie has the following goals she hopes to
achieve:
• become an effective MPS Society legislative
advocate for MPS and related diseases
• further promote member involvement and
education in the legislative process
• support family fundraising and awareness
events
• encourage creative approaches to successful
fundraising
• provide peer support to all members coping
with the challenges of living with MPS and
related diseases
• provide compassionate support to newly
diagnosed families
Hope and Dave Madsen reside in Lakeland,
MN, and are parents of three young adults: Jake,
24; Ben, 22; and Fran. Fran, who has MPS I, is
a 21-year-old electrical engineering student at
Michigan Technological University. Dave has
a master’s degree in optics from the University
of Rochester and is employed as an optical
engineer at Rudolph Technologies and an
continued
“With our children well on their way to being
independent adults, we thought it was a good
time to really get involved with the National MPS
Society and help out in any way possible,” said
the Madsens.
The Madsens founded and chaired the 2010
Minnesota MPS Cup with the Minnesota NHL
alumni and will host the 2011 Minnesota MPS
Cup. They hope “to dig into a committee and
produce some tangible improvements over the
course of the next two years.”
Dave, Hope and Stephanie are all involved in
their communities, working with their schools,
churches and serving on community boards.
Jeff Bardsley is a young adult with MPS II
who resides in McLean, VA. He has an older
brother who, with his wife, lives in the San
Francisco area. Jeff completed his bachelor’s in
business administration in 2009 and master of
accountancy in 2010, both from the University
of Richmond, and works as a business analyst for
a government contractor in Reston, VA.
New Board Members
adjunct professor at the University of St. Thomas.
Hope has a bachelor’s degree in mechanical
engineering from Michigan Technological
University and is employed as a mechanical
engineer at CyberOptics.
“I wanted to become more active in the MPS
community because I believe fellow affected
young adults have the potential for tremendous
contributions and future leadership in the MPS
Society,” said Jeff. “I believe the Society could
benefit from having a direct patient perspective
on the board.”
Jeff’s ultimate goal is to increase public awareness
so living and caring for an MPS individual is less
difficult, and so that a new generation of young
researchers are drawn to work on all forms of
MPS diseases so that ultimately there will be a
future where patients have different treatment
options available and possibly a cure.
Family Support Committee Announces New Program
Medical Travel Assistance Program assists families with long-distance
medical appointments
Families may need to travel long distances to consult with medical professionals who are knowledgeable
about MPS and related diseases. The Medical Travel Assistance Program (MTAP) helps fund out-oftown travel costs for such non-recurring medical appointments. MTAP may reimburse up to $500 per
affected individual, per 12-month period, in transportation costs for member families traveling to a
medical appointment more than 200 miles from their home.
Reimbursable expenses include air, train or bus fares, rental car, airport parking, and transportation to
and from the medical facility or appropriate mileage at the current IRS rate. Only coach airfares are
eligible for reimbursement. A copy of the ticket invoice and other original travel receipts are required
for reimbursement. Verification of the attended medical appointment must be included. Please read
program guidelines in the Family Support section of our Web site for complete details.
The MTAP is a three-step process. In step 1, the applicant will submit an application to request funding
for a future appointment. The Society will notify applicant if funding has been approved. In step 2,
the approved applicant will attend the medical appointment and have the verification form signed by
the medical professional. In step 3, the approved applicant will submit the completed reimbursement
request and verification forms along with the travel receipts to obtain reimbursement.
To request an application or if you have any questions, contact LaurieTurner at 207.843.7040 or
[email protected]. Applications also can be downloaded from the Family Support Section of the
MPS Society Web site.
13
OSCAR Awards
Outstanding Sibling, Children and Relatives (OSCAR) Award
We are excited to present a new section of Courage to honor the brothers,
sisters, children and relatives of our children with MPS and related disease. This
special group of individuals provides support, humor, direction and most of all
unconditional love. We honor super siblings who shine each and every day. To
nominate someone in your family for an OSCAR award, please send an e-mail
to Laurie Turner at [email protected]. Names will be selected randomly to
be featured in each issue of Courage.
Courtney Sanford
Courtney Sanford’s sister, Cara, lost her fight with MPS III on Dec. 27, 2010, at the age of 17. Courtney
was and will always be the best big sister. She loved Cara unconditionally and with no regrets. The best
way to describe that love is in this poem that Courtney wrote when she was a senior in high school.
With Love Always
It’s just so sad.
I’m living the life
That you’ve never had.
Time goes by, day after day,
Hoping and praying that you’ll always stay.
No one will ever know this guilt and regret,
Thirteen years and still I feel we’ve never truly met.
Cara (MPS III) and Courtney
Sanford
The words, they’re always so hard to find,
Don’t you know I’d give anything,
To know what’s on your mind.
I want to get to know you,
I wish you’d talk back.
Never hearing your voice,
Something we’ll always lack.
This wasn’t the way it was supposed to be,
You were supposed to grow up,
And get old with me.
But the way you are,
It’s the only thing I’ve ever known.
I wouldn’t change you for the world,
And I’m proud to be able to call you my own.
You know I’m here for you until the bitter end,
With love always, my sister,
My friend.
14
Following is an excerpt from an article that appeared in the San Angelo Standard Times, Dec. 21, 2010.
Written by Rick Smith. To read the complete article, go to www.gosanangelo.com/news/2010/dec/21/sick-boysparents-hold-on-to-hope/.
Sick Boy’s Parents Hold on to Hope
In many ways, he’s like any other young boy. He loves dogs, Nintendo DS, sports and SpongeBob. He
likes spaghetti and ice cream. He draws great pictures. He wants to be a police officer or a soldier
when he grows up. But Shawn Chavez, who turns 8 years old Feb. 1, has a rare medical condition. His
parents worry time may be running out for their special boy.
Annette and her husband, Chris, moved from McCamey to San Angelo two months ago in hopes of
finding answers for Shawn’s disease. Mucopolysaccharidoses (MPS) is a genetic disorder that occurs
in one out of every 200,000 births. There is no treatment for the problem, though some symptoms,
ranging from heart problems to cloudy corneas, can be treated as they occur.
Making Headlines
One of the goals of the National MPS Society is to increase awareness of MPS
diseases. With the assistance and persistence of our members, we are making
great strides. Don’t forget to let the MPS Society know when you are featured
in a media story!
Following is an excerpt from an article that was posted on Apple Valley Patch, a Web site serving the community
of Apple Valley, MN, Jan. 18, 2011. Written by Austin Cumblad. To read the complete article, go to http://
applevalley.patch.com/articles/the-biggest-little-man-on-campus.
The Biggest Little Man on Campus
Michael Sheridan’s experience at Eastview High School should seemingly be controlled by his rare
metabolic disease. Instead, it’s defined by his love of basketball and the school’s love for him.
Michael Sheridan stands alone on the basketball court after school, 10 feet from the hoop. He takes
his first shot; it clangs off the rim. He tries a layup; it bounces out. Then suddenly, he can’t miss. He
makes two jumpers. And two more. Five-for-seven, six-for-eight.
Now it’s time for practice. Michael stops shooting, dutifully rounds up the balls and puts them back
on the rack while the Eastview High School girls’ basketball team stretches. He’s the team’s manager.
Even though Michael shoots 75 percent from the field, he will never play varsity basketball. But
Michael loves the game and the coaches and the team. And they all love him back.
Wish Come True
Luke Chambers (MPS I) recently was featured in Hot Wheels magazine when the National Hot Rod
Diesel Association and the Dallas, TX, chapter of the Make-A-Wish Foundation teamed up to give five
kids and their parents passes to the Texas Diesel Nationals race. The children were given VIP, behindthe-scenes treatment, including meeting drivers. Luke was even able to take a ride in a truck on the
track!
15
Annual Fund
Annual Fund Campaign Raises $85,591 in 2010
We reached a new record of giving in 2010—an amazing year for the Annual Fund! This critical funding
stream within the Society keeps the organization strong. Through your tremendous support during
difficult times, you have shown the Society you understand the need of our families and those affected
by MPS and related diseases. Your gift will provide support to families through the Family Assistance
Program, provide funding for federal advocacy initiatives and provide necessary operating funds.
This year the board of directors reached out with phone calls and spoke to our membership and
friends of the Society. We are so encouraged by the conversations we had with your families. It was a
terrific opportunity to say hello and thank you for supporting our efforts.
For everyone who reached into your pockets once again and donated to the Annual Fund in 2010, the
Society gives our sincerest thanks!
Philantropist
$1000 or more
Raymond and Barbara Alpert Foundation
Mel and Millie Anhalt in honor of Conner Anhalt
Wayne Bardsley
Vaughn ad Nancy Bryson
Joe and Kelley Capone
Joan Cook in honor of Jacob and Samantha Slawson
Ernie and Debbie Dummann, duAlaska, Inc.
John Exner in honor of Jayden and Brooklyn Bryce
Family of David Downing
in memory of David Downing
Stephen and Betsy Fowler
Eileen Gideon
Scott and Lynn Hopkins
in honor of Michelle Hopkins
Wayne and Lori Hummel
Kevin and Tracey Litwin in honor of Jack Frye
Robert and Marjorie Lovell
in honor of Olivia Lovell
Jan and Joe Melnyk
Mark and MaryEllen Pendleton
in honor of Allison Kirch
Crystal Polis
Pool Tool Inc.
George and Athena Sarantinos
in memory of Jennifer Wingate
William and Ann Stark
Tracy Szemanski
in memory of Clinton and Zachary Szemanski
Amy and Klane White
in memory of Susannah White
in honor of Scotty Whitecotton
Leader $500–$999
16
Art and Judith Ackerman in honor of Jack Frye
Frederick and Nancy Andrews
Brenda Baker in honor of Jack Mahony
Joel, LeeAnn and David Bernbaum
in memory of Mark Bernbaum
Lorraine and Frank Bien in honor of Cecilia Bien
Richard Bosse in memory of Dorothy Bosse
Diane and Rob Cassil in honor of Matthew Cassil
Larry and Margaret Cohen
Ralph and Jane Daniels
in honor of Branden Patterson
Stephen and Kimberly Frye in honor of Jack Frye
Sharon Glasscock in honor of Taylor Geary
Steve and Amy Holland
in honor of Spencer, Maddie and Laynie Holland
Mark Hopkinson in memory of Mark Bernbaum
Wendell and Karen Keith
in memory of Amanda Keith
Larry and Susan Kirch in honor of Allison Kirch
Joseph McCarthy in honor of Jack Mahony
Carlos Otero in honor of Carlo A. Otero
Larry and Mary Sweeney in honor of Denise Dengel
Donald and Lisa Wells in honor of Matt Wells
Gordon Wingate in memory of Jennifer Wingate
Volunteer $250–$499
Mary Starr Adams in memory of Paul Adams
in honor of Conner Anhalt
Sandra Babel in honor of Danny Miller
Shawn Barkley
B.L. Bickham
Cheryl Bien in honor of Cecilia Rose Bien
Scott and Peggy Dionne
Charles and Josephine Ellard
in honor of Karina Guajardo
Bart and Sally Finzel in memory of Eddie Ducham
Tom and Anne Gniazdowski
in memory of Danny Gniazdowski
Wallis, Monica, Natalie and Nicole Hampton
in honor of Jake Hampton
Mark Haskins
Peggy Holland
in honor of Spencer, Maddie and Laynie Holland
Barbara Ives in honor of Mackenzie Grace Clarke
John Hancock Foundation
in honor of Bryce Chesser
continued
Friend $100–$249
Dean and Kathy Aker in honor of Sam Aker
David and Ada Alden in honor of Jack Mahony
Tim and Mikki Annis in honor of Stephanie Annis
David and Stacey Aronson
in honor of Mitchell Finzel
Stephen Aselage
B.D. Ashe in honor of Kayla Martin
Mark and Alberta Balliet
in memory of Richard and Jennifer Balliet
Joseph and Jacqueline Balukin
in honor of Michael Bodura
Jane Barker
Debbie and Dwight Barkley
in honor of Davis Barkley
Patricia Baxendale
in honor of Molly Birmingham
Big Hook Crane Service, Inc.
in memory of Stephen Weaver
Jennifer and John Birmingham
Tara Ann Blazer
in honor of Jayden and Brooklyn Boyce
Mike and Grace Bodura
in honor of Michael Bodura
Tom and Diana Boland in honor of Aspen Knight
Marie Bonville
Katherine and Joseph Bonville
Marsha and Robert Bozarth
in honor of Annabelle Bozarth
Donna Brinkley in memory of Spencer Holland
Kathryn Bullard in memory of Michael Zechender
Nancy and Paul Burke
Christina Byrne in memory of Gabriella Giannone
Michael and Margaret Ciacciarelli
in honor of Max Ciacciarelli
Steven and Jennifer Clarke
in honor of Mackenzie Clarke
Kent and Karlene Classen
in memory of Karlee Rae Classen
Ross and Belia Coddington
in honor of Karina Guajardo
Esther Conrad in honor of Joshua Williams
Michael and Claire Constantine
in memory of Chris Rousseau
William and Peggy Cook in honor of Daniel Miller
Brooke and Stephen Cornwell
Bob and Laura Cowin
Jeremy and Billi Crockett
in honor of Matthew and Camden Crockett
Elizabeth Demkin
Ray Dettmer
B.H. and Barbara DeVries
Erline Dickerson
Jonathan and Lisa Dois in honor of Nathan Roma
William and Bonnie Doster
Phil and Donna Dudley in honor of Rylie Hays
Carla Ellard in honor of Karina Guajardo
N. Matthew Ellinwood
Tara and Jack Elston in honor of Jake Elston
Mike and Kathy Epstein
in honor of Gerald Anhalt’s birthday
Patricia Farquhar-Morris
Robert and Amy Farrell
in memory of Joe and Maggie Langford
Pamelia Farthing in honor of T.J. Catanzarite
Phyllis Feibelman
in honor of Petey Feibelman’s birthday
Gail Finney in memory of Clinton, Zachary and
Larry Szemanski
Rod and Kathy Finzel in honor of Mitchell Finzel
Donald and Ramona Finzel
Sarah and Greg Fletcher in honor of Erik Fletcher
Lennie Forkas in honor of Scotty Whitecotton
Jason and Jamie Fowler in honor of Jack Fowler
Annual Fund
John and Janet Kappel in honor of Debbie Kappel
Alan Kimura
Terri Klein
Brian and Kristine Klenke
Joel and Lisa Klessens
in memory of Morgan Klessens
Laurel Creek Hardwoods, Inc.
in honor of Taylor Geary
Lori LeDoux in honor of Matthew Caldwell
Robert Harvey and Roxanne Maffitt
Sunni and Gary Markowitz
Maria Meconi in honor of Denise Dengel
Elizabeth Neufeld
Austin and Dorothy Noll
in honor of Austin Noll IV
Joseph Orendain in honor of Ganesh Shrestha
Alan and Diana Pendley
Ewa Robinson in honor of Jack Mahony
Naureen Sayani
Tami Slawson
James and Sheila Slawson
John and Diane Stabb in honor of Danny Miller
Darlene Sweeney in honor of Denise Dengel
Robert Maiden and Becky Tanamachi
Terra Properties, Inc. in honor of Kraig Klenke
David and An Tootill
Jon and Eleanor Totz
Beth Valocchi in honor of Ryan and Brayden Kapes
Kenneth and Barbara Velten
in honor of Steve and Bryce Chesser
Barbara Wedehase
Stuart Swiedler and Judy Weiss
Hubert and Elaine Willman
17
continued
Annual Fund
18
Donald and Mary Jane Fowler
in honor of Kimberly Fowler
Mark Czaja and Joyce Fox
Ronald and Sheila Francis
Timothy and Roslynn Garvin
in honor of Marcus Garvin
Kevin and Andrea Gates in honor of Spencer Gates
Patricia Giles
in memory of David, Randy and Jared Barrett
Thomas and Jean Gniazdowski
Russell and Catherine Goodale
Dan Gotel
Blake and Ashley Graeber
in honor of Bryant B. Graeber
Rosemarie Greco
Leonard and Phylis Greenstein
in honor of Zachary Townsend
Luis and Angela Guajardo
Sheryl and Gerald Hall in honor of Julie Hall
Shelby and Tim Haslett
in memory of Douglas Sampson
Shawn Healy in memory of Scott Healy
Keith Hess
Christine Ho in memory of Susannah White
William and Margaret Hughes
in memory of Kristopher Arnold
James and Mary Kalteux
Tom Kirch in honor of Allison Kirch
Fred and Joyce Koehler in memory of Ryan Mask
James and Norvella Koelling
Andrew Kramer in honor of Davis Barkley
Douglas Kreul
Lancaster Lions Club in honor of Jeff Stribing
John and Joyce Lane in honor of Trey Lane
Philip and Dawn Laorenza
in memory of Aurora Laorenza
David Lebec in honor of Michael Bodura
Ron and Monica Leone
in memory of Kristofer Arnold
Catherine Lewis
Jaime and Jennifer Lipscomb
in honor of Olivia Lipscomb
Lloyd and Cheryl LoCascio
in memory of Logan LoCascio
Gene and Cynthia Logan
in honor of Rhianna Logan
Michael Longo
Richard and Mary Losee
Chuck and Nancy Lukondi
Barbara and George Malina
in honor of Glen Malina
Mike and Carolyn Managan
in honor of Ricky Hodgkins
Eileen Marchica
Frances McCauley in memory of Paul Adams
Stephen and Marie McClurg
in memory of Dylan Michael McClurg
Ward McKeithen in memory of Edie Burke
Raymond and Amanda McPartlin
Mary Ann Messick
in honor of Russell and Alexander Browne
Donald and Joan Miller in honor of Danny Miller
Gary and Carol Moll in memory of Christopher Moll
Mitchell and Cherylynne Moore
Bernard and Evelyn Morgan
Richard and Eva Morgan
Ron and Chris Morrissey
in memory of Cade Morrissey
Gary Mortensen
Rene and Patricia M’Sadoques
in memory of Anthony M’Sadoques
Denise Mullady in memory of Patrick Trainor
Carol Nareski
Anne Novak
Ronda O’Donnell
in honor of Kaitlyn and Sadie O’Donnell
Margaret Offutt in honor of Marcus Garvin
Thomas and Barbara O’Malley
Ann O’Malley in honor of Allison Kirch
Dr. and Mrs. Anthony Pisa
Thomas and Sharyln Pope
in honor of Lauren Pope
Flossie Pryor in honor of Andrew Cochenour
Sue Rattman in memory of Adam Rattman
John and Debby Ratto in honor of Maria Ratto
Bernard Ray in memory of Peter Wehrle
Chip and Wendy Rennaker
in honor of Mindy and Jill Rennaker
Gary and Bonita Ritondaro
in memory of David Hedrick
Sherrie and Tony Roman
Hickman and Carey Rowland
James Kremers and Sally Rullman
Dr. and Mrs. A.L. and Angela Sarantinos
in honor of Luke Sarantinos
Arthur and Marilyn Sheekey
in memory of Laura and Michael Sheekey
Mary and Thomas Shine
Alberta and William Stanley
Meghan and Billy Stark
Peter and Sarah Storlie in honor of Allison Kirch
Morton and Susan Stuhlbarg
in honor of Bryce Chesser
James and Carolyn Stultz
in memory of Maggie and Joey Langford
Kim and Bill Swartz in memory of Alex Sobie
Steven and Lynn Szydel
Hal and Laura Thorsrud
in honor of Julie Thorsrud
Eleanor Tobin in honor of Michelle Hopkins
Carol Treutlein in memory of Stephanie DeAngelis
Laurie and Eric Turner
continued
Donor $26–$99
Frank and Nancy Aiello in honor of Katie Shine
George and Gail Anderson
Ralph and Martha Anderson
Richard and Carol Anderson
Krista Baker in memory of Emma Turner
Gary and Debbie Ball
Leonard and Barbara Barcousky
Amy Barkley in honor of Davis Barkley
Donald and Nina Bergquist
in memory of Mark Bergquist
Bob and Eve Bonville in honor of Allison Kirch
David and Stephanie Bowser
in honor of Maddie Wigglesworth
Julie and Joseph Brackett
Clarence and Faye Brown
Gary and Karen Brugnoli in honor of Taylor Geary
Don and Frances Bryant
in honor of Bryant Graeber
Nancy Bucher in honor of Tyler Seabrook
Kim and Kevin Burke
John and Barbara Casino
Heidi and Keith Caswell in honor of Sam Caswell
Denise Caswell in honor of Sam Caswell
Anthony and Donna Cataldi
in honor of Cara Sanford
Shirley and Tony Catanzarite
in honor of T.J. Catanzarite
Steve and Karole Chesser
in memory of Don Westerland
Kayla Cotkin
in memory of Elliott and Martin Cotkin
Allen Crocker
Estelle Culpepper in memory of Geoffrey Anders
Steve Cuomo in honor of Trey Lane
Mike and Lisa Day
in memory of Greg and Scott Day
Jessie DeHaan in honor of Livia Hubert
Darrell and Kay DiStefano
in honor of Logan Piefer
Janice Dittmar in honor of Kaitlyn O’Donnell
Jeffrey and Mary Dozbaba
Lou Fabian in honor of Cindy and Ricky Fabian
Karas family in memory of Mark Bernbaum
Jacqueline A. Fenaroli in honor of Austin T. Nace
Patrick and Patricia Ferreri
in honor of Rachel and Jessica Hess
Maria and Steven Foshay
in memory of Katie Foshay
Anna Funkhouser in honor of Davis Barkley
Nic McPhee and Susan Gilbert
Lee and Tammy Gottschall
Marie Griffin
Stanton and Jean Gunnells
in honor of Daniel Jones
John and Cheryl Hackett
in memory of Maggie and Keegan Hackett
Mick and Shirley Hagermann
in memory of Tom Haggermann
Natalie Haggett in honor of Zachie Haggett
Steve and Cynthia Hamblen
Rodney and Donna Hardy
in memory of Adam Hardy
Jeffrey and Barbara Harrell
Lena Harris in memory of Greg and Scott Day
Jim Haefemeyer and Susan Hoch
in honor of Jack Mahony
Linda Horn
Todd and Jennifer Howard
in honor of Joseph Delvaux
Robert and Marguerite Iannacone
Rick and Jeanne Joiner in honor of Riley Joiner
Kathleen Jones in memory of Jamie Drummer
Heidi Kanealy
Susan Ketchum in memory of Robert Grisham
Henry and Ardis Ketterer
in memory of Peg Ketterer
Jennifer Klein
Mr. and Mrs. Allan Krech
Darren and Patty Latham
Ken and Sandy Ledford
in honor of Amanda Crawford
Joanne and Bill Lennon
in memory of Martin Elliott Jr.
Linda Jean Cohen Levin in honor of Sydnee Jensen
Scott and Gabriella Lich
Jack Lowe in memory of Joseph Raftery
William and Alayne Lowenberger
in honor of Michael Bodura Jr.
Amy Mauldin in honor of Jessica James
continued
Annual Fund
Helen and Bill Vespe
in honor of Mary and John Hoffman
Mary and Jeff Von Handorf
Rex and Sharon Wang in honor of Shihjay Wang
Kathy Ward in honor of Bryce Chesser
Kenneth and Victoria Warner
in memory of Bradley Chapman
Way Elementary School Social Committee
in memory of Sandra Christine Webb
Claude and Roselyn Wells
in honor of Matthew Wells
Gary and Tracy Wells
John Williams in honor of Jeffrey Orr
Lynn Ann Witt
19
Annual Fund
20
Mary McFarland in honor of Maggie Rousseau
Judith McKinstry in memory of Adam Hale
David and Christina Michaelmore
Glenn and Kathy Miller in memory of Glenn Miller
Richard and Debra Moran
in memory of Greg and Scott Day
Tom and Sheila Morley in honor of Payton Morley
Jim and Paula Moskal
Soraida Munoz in memory of Tara Matthews
Michael and Anita Muonio
in memory of Joseph and Zachariah Muonio
Susan Narducci in honor of Danny Miller
Raymond and Julie O’Hara
in memory of Kristopher Arnold
Mary Page in memory of Danny Gniazdowski
Anthony and Pamela Paladino
in honor of Caden Mitchell
Florence and Mario Paolilli
in honor of Austin Nace
Elizabeth Binkley and Robert Park
Ronda Paullin
Teresa Peecook in honor of Lynn Peecook
Tom and Carrie Porzel
in memory of Robert Alvarado
Lawrence Pung in honor of Austin F. Noll IV
Stephanie and Edmund Rabuse
in memory of Paul Adams
Janet Rafferty in honor of Shannon Tootill
Floyd Reed in honor of Evan Reed
Stella Reeve in honor of Kristen Reeve
Lindsey and Karen Rice
in memory of Christopher Migliozzi
Shawna Robson in honor of Jaeda Robson
Isabella Rodrigues in memory of Diana Rodrigues
Mr. and Mrs. A. Kay Rogers
Rosemary Rotelli in honor of Richard Rotelli
Suorchi and Thavy Sasry
in memory of Samantha Sasry
Mark Schlafer
Betty Shade in honor of Tyler Seabrook
Judy Shafiee in honor of Sharon Browne
Greg Sharrits in memory of Edie Burke
Loretta Shaw in honor of Sydnee Jensen
Muhanad Jawad and Jumana Shawkat
in honor of Zayd Jawad
Robert and Ann Sheridan
in honor of Michael Sheridan
Burt and Eileen Skolnik
Robert Slowey
Julia Smith in memory of Greg and Scott Day
Sergio and Angela Stasi in honor of Erin Peters
Jeffrey Stribing
Donald and Rachel Swicker
in memory of Jon and Joe Karas
Allen and Jackie Theriot
in honor of Blake and Lance Dutile
Fern Titus in honor of Waverly and Oliver McNeil
Nita Todd
Ruth and Matt Travis
James and Bonnie Tully in memory of Paul Adams
Harry and Janice Van Horne
in memory of Rishi Garg
Rosemarie and Edmundo Veliz
in honor of Chase Veliz
Steve and Martha Waters
Ruth and James Watkins
in memory of Andrew Watkins
Claude and Roselyn Wells
Joan Werner in memory of Mark Bernbaum
Dr. Don Westerland in honor of Bryce Chesser
Pamela and Darrell Whittle
in honor of Mason Whittle
Raymond Young in memory of Kaylee Kacer
Patron $5–$25
Bob Ahern
Betty and Barney Arceneaux
in honor of Braden Farrell
John and Janet Arendt
in honor of Michelle Hopkins
Kim Armstrong in honor of Bryce Chesser
Sally Armstrong in honor of Logan Piefer
Sherri and Scott Baggett
Wade V. Baise in memory of Erich Nikolas and
Garrett Curtis Baise
Mary Ellen Barringer
in memory of Zachary and Clint Szemanski
Rose Ann and Dominic Bencivenga
Todd and Nicole Blancheri
in honor of Wyatt Blancheri
Robert Brulhardt Jr. in memory of Paul Adams
Tony and Claudia Busse
Saundra and Larry Clark
in memory of Devon Clark
William and Peggy Cook in honor of Danny Miller
Diane McGuire Cooper
in honor of Kallie Alyssa McGuire
Bob and Denise Crompton
in memory of Kelley Crompton
Marjorie Ditmore
Geri Edwards in honor of Danny Miller
Darla Elswick
Wlbur and Irene Erhardt in honor of Matt Wells
Peter and E.F. Fairchild
Helen Fallon in honor of Michael Bodura Jr.
Barb Fisher in honor of Allison Kirch
David and Leesa Galloway
John Gladysz in memory of Danny Gniazdowski
Jennie Gladysz in memory of Danny Gniazdowski
Dennis and Christine Goggins
in honor of Sam Anthony
Michael and Frances Goldberg
Arthur and Brenda Hawkins
Roy Hedrick in honor of Hanna Taulton
Marlys Herring
continued
Eugene and Elizabeth O’Shea
in memory of Patrick Trainor
Larry and Eileen O’Steen
in memory of Ryan O’Steen
Sandra Page in honor of Marcus Garvin
Patti Pair in memory of Audrey Lawson
Dave and Michelle Patterson
Shirley Pelletier in memory of Amanda Keith
Ambrose and Patricia Perreault
in honor of Logan Marcotte
Douglas and Mary Pica
Katy and Jay Plotkin
John and Mary Ratto in honor of Maria Ratto
George and Carol Rice
Mr. and Mrs. W. Frank Rogers Jr.
in memory of Chris Myers
Barbara Russo in memory of Amanda Kieth
Donna and Thomas Schidlmeier
in memory of Jaret Schidlmeier
Lowell and Janet Schmidt
Donald and Janet Schmidt
Jody and Joel Shapiro
in memory of Joe and Maggie Langford
Ron Shebik
Nancy and Kent Shelton in honor of Allison Kirch
Shirley Smith in honor of Kyle Lingo
Myrna Stelman in honor of Jack Frye
Deborah Toga in memory of Chad Pyper
Jean Jacobsen and Richard Underwood
Barb and Mike Weikel in honor of Katelyn Vanghle
Lewis Whitaker Jr. in honor of Logan Piefer
Richard and Margaret White
in memory of Richard White III
Rick and Rebecca Wildman
Minnie Wood
Andrew and Louise Zygmuntowicz
Annual Fund
Wayne Howser in memory of Jenny Wingate
Janice Inman in honor of Jim and Tim Oliger
and Bil Selmeyer
Lisa and Chris Jones
Gloria and Irwin Katz in honor of Jack Frye
Charles and Sandra Kegel
in memory of Kylie and Kendall Moran
Howard Kelfer in memory of Cameron J. Green
Millicent and Charles Kennedy
in memory of Russell and Dougie Kennedy
Gordon and Nancy Kinley
Janet and Gary Kirch
Herbert Kirch in honor of Allison Kirch
Allen and Mary Jane Koehler
Louise and Edward Kofron
Victor and Pamela Kovach
in honor of Alleis Wilson
Clint and Nikki Kremer in honor of Ava Kremer
Lynn Lilore
Norine and Thomas Lippens
in memory of Cade Morrissey
Edward and Suzette Ludloff
David and Regina Marcotte
in honor of Logan Marcotte
Richard and Janice Marino
Fay Matthews in memory of Tara Matthews
James and Eleanor McCaffrey
Jonathan and Christine Mclain
in honor of Julia Mae Valentine
Larry Michael in honor of Lauren Pope
Pauline and Robert Michek
Erna Mlekusch in memory of Paul Adams
Merritt and Madeline Moseley
in memory of Edie Burke
Bernadine Nardin
Robert and Marilyn Nehus
in honor of Helen Bodura
David and Frances Notley
in memory of Nancy Notley
Billie Sue Nutter in honor of Davis Barkley
Did You Know?
May 15 is International MPS Awareness Day
Each year the Society celebrates International MPS Awareness Day on May 15. This day is
devoted to raising awareness of MPS and related diseases, and is a way to honor everyone
in the MPS community. MPS Day offers an opportunity to remember all the children and
adults who suffer from MPS and related diseases; to think about the children we have lost; to
recognize the doctors and scientists who are dedicated to finding a cure; and to remember
each other and be thankful for the strength and support we both give and receive.
Many families celebrate this day by hosting a fundraiser, wearing purple, having a balloon
release or simply thinking of or remembering an MPS child. How will you celebrate?
21
Family News
YOU ARE
IMPORTANT
TO US, PLEASE
KEEP IN TOUCH.
Please remember
to let the Society
know if you are
moving. In addition
to helping keep
printing and postage
costs down, you’ll
help us keep our
database up-to-date.
Keep us informed
of new addresses,
telephone numbers,
e-mail addresses, and
any interesting news
about your child.
22
I hope that all is well with you and you had a good 2010. We
had a good year overall. Our twins, Hannah and Luke, started
kindergarten which was rather exciting. We also went on Jake’s
Make-a-Wish trip to Disney World in November. That was a lot
of fun, however Jack and I came home feeling like we needed
a vacation from our vacation. It was rather exhausting.
Jake continues to do well. He has some three- and four-word
combinations but certainly does not have conversational
speech. He is able to let us know what he needs and understands
what we want or need him to do. He turned 4 in January. It
is hard to believe that next week we are already coming up
on the two-year anniversary of his diagnosis. Aside from a few
random nights of sleep issues, overall Jake is doing great.
Tara Elston
Jake with Pluto at Disney World
Mia (MPS III) had a service dog for 11 years named Emily who passed away in 2009. With our move
to Ohio, we decided it was time to get Mia a new dog to help her in all the ways we could not. I was
elated to receive a phone call from Circle Tail, which provides service dogs and hearing dogs to
people with disabilities. I really liked Breno, but I wanted it to be Mia’s choice. Thankfully, he was
such a well-mannered dog and impressed Mia with all his commands and tricks that she wanted him
too! He is a totally different dog, but better in ways I cannot even tell you. This dog is ready to gogo-go! He rides everywhere with us. He walks right next to her wheelchair on our marathon walks.
Mia feels like a very important girl when people ask her if they can pet her dog. He wants to be in
the room with us all the time, he makes Mia laugh when he snores, he will do all his commands and
tries to make some up when she has a treat for him.
Since Breno moved in, he amazes us every day. He has even tried to help me pull laundry out of the
washer and open the front door. He waits on the porch with me for Mia’s bus, and sometimes walks
up into the bus if she is taking too long to get off. He waits by the door when it is time for her bus
to come in the morning. If he is downstairs with us and she calls him upstairs, he takes off up those
stairs! Mia is so proud that he is doing all of these things.
What a welcome for a lonely kid who needed this dog more than we even realized.
Mia, Billy and Kimberly Pruett
Circle Tail can be contacted at:
8834 Carey Lane, Pleasant Plain, OH 45162
513.877.3325
Lucas (MPS III) and Stacey Montgomery
Livia Hubert (MPS III) with mom Kelly,
dad Jake and sister Finley
Surprise! It’s a boy! After four years, Ryan’s birth
mother delivered a baby. She hoped we would
adopt him so that he and Ryan could grow up
together. On April Fools Day, five days later after
emergency custody papers were signed, we took
Jacob Patrick Moore home with us. He was the
perfect Easter gift. We still had lots of legal issues
to finalize but after seven months we were able to
adopt him (on Jessica’s birthday).
Amazing! We decided to host a benefit at
Davanni’s Pizza for Jessica’s disease, MPS I.
Hundreds of people came to show their support.
It was a huge success and proved that she is a
very inspirational girl. She had knee surgery
in January and with therapy has had an active
year—skiing, swimming, choir, theater, scouts
and she turned 13 years old, officially a teenager.
Movies, dances, lock-ins, sleepovers, homework
and hormones make never a dull moment in our
house and yet she is a beautiful young lady and a
great big sister.
Score! Jason has been playing hockey on a men’s
league. When we were approached by MPS
friends about putting together an MPS hockey
benefit where he would get to play some of
the NHL alumni greats, we were all for it. We
planned the gala event and raised thousands of
dollars for families with MPS. There was a hockey
game during the day and dinner, auction and
drinks at night. Both the MPS Society and the
NHL praised our efforts, so we’ll host the event
again this year and Colleen will be the speaker.
Mark your calendars for April 16.
Encore! Jessica and Colleen were in the St.
Odilia Church musical production of Honk!
It was the ugly duckling story and there were
many adaptations to include diverse children
with special needs. Jessica was selected for one
of those roles because she knows first-hand that
different is just different, not good or bad.
Jason and Jon (MPS III) Vanderpool
Yahoo! Ryan turned 5 and is in kindergarten.
He loves school, especially the bus ride. Every
day he comes home with sound and sight words.
He has been reading and will write notes (some
need explaining), has a great vocabulary, colors
pictures and really likes to learn. He has always
been active but this year he discovered his twowheeler, or should I say dirt bike. He was riding
everywhere and creating jumps. He enjoyed
soccer camp, scout camp, hockey, swim lessons
and Jason was again his t-ball coach. He likes
to hang with the big boys and is always up for a
game of cards. He loves being a big brother and
will announce Jacob likes him best since they are
really brothers.
Family News
What a year it has been in our house. Things
change every minute without notice. We were
looking back on the year and many words have
come to mind.
Whew! Jason has been working for FAF Advisors
which is a subsidiary of USBank for 10 years; it
was recently sold to Nuveen Investments. He
was one of the few employees to keep his job. As
of the new year he will be doing some different
tasks for USBank but in this market he is thrilled
to have a job. We had our house for sale but
opted to stay where we are since Colleen is not
currently going back to work but caring for a
new baby instead.
So big! Jacob has done everything backwards
from the minute he was born. We figure we get
nine months to adjust to having him around. He
was born, and then we found out. He is sitting
but still doesn’t roll over. He refused baby food,
went from six months of the bottle to table food
within weeks. He loves spaghetti and like a true
Minnesotan, hot dishes. Maybe it’s because he
was 10 lbs. when he was born. He got his corner
teeth before the front ones and is on the verge
of pulling himself up and crawling. Jacob’s
giggle would put Elmo to shame but he also has
mastered the dead stare. It is fun having a baby
around again.
Hope you have a very wonderful 2011, full of
adventure and blessings. As for us, we can’t wait
to camp next summer and see some beautiful
national parks on our way out to Oregon.
Jason, Colleen, Jessica, Ryan and Jacob Moore
Hopkins family
23
Holiday Photos
2
1
4
3
5
7
6
8
11
9
12
10
13
14
1
Alena Galan (MPS IV)
2
Nicole and Jack (MPS II) Frye
3Luis, Angela, Anyssa and
Karina (MPS III) Guajardo
4
Haley and Danny (MPS II) Miller
5Jamie, Conner (MPS II), Cynthia
and Randy Anhalt
15
6
Andrew and Sydnee (MPS I) Jensen
7Allison (MPS I) and Ashley
Restemeyer
8
16
Carter and Makenzie Bays
9Annabelle (MPS IV) and Madeline
Bozarth
10Matt, Shannon, Oliver (MPS III)
and Waverly (MPS III) McNeil
11 Mason Whittle (MPS III)
12 Jenna and Sam (MPS I) Caswell
17
13 Blair (MPS III) and Grey Chapin
14Ella, Zachary and Mackenzie
(MPS III) Clarke
15Laura, Bob, Will (MPS II) and
John (MPS II) Cowin
16Chris, Mercedes, Ben, Alex, Dorian
(ML II) and Wynn (ML II) Johnson
24
18
17 Braden (MPS I) and Megan Farrell
18 Shelby George (MPS III)
Holiday Photos
21
19
20
24
25
23
22
27
26
28
29
19Jason, Christi, Logan and
Levi (MPS III) Ormeroid
30
32
20Jason, Jamie and Jack (MPS II)
Fowler
21 Elston family (MPS III)
22 Zain Semones (MPS II)
31
23Steve, Laurel, David (MPS II),
Chip (MPS II) and Amy Radius
33
24 Durcholz family (MPS III)
25Shane, Jenifer, Clara (MPS III)
and Ethan Gibson
34
26 Wojnarowski family (MPS III)
27Jake (MPS II), Natalie and
Nicole Hampton
28Jonathan, Marie, Adam, Jana
and Ryan (MPS II) Hunt
29 Helen and Allison (MPS III) Kirch
30 Luke Chambers (MPS I)
31 Klenke family (MPS II)
32Karen, Lindsey (MPS III) and
Hannah Efird
35
36
33 McDermott family (MPS III)
34 Moore family (MPS I)
35Kassi (MPS III), Mike and Tyler
Offenbacker
36Evan, Luke (MPS III) and George
Sarantinos
25
Fundraising News
On Nov. 20, 2010, the Pi Kappa Alpha fraternity completed a 440 mile roundtrip bike-a-thon from
Rock Hill, SC, directly to the National MPS Society office in Durham, NC.
The fraternity has a bike-a-thon each year to benefit a cause. After contact with the Isabel Juardo
Foundation in Rock Hill, SC, they suggested raising funds for the National MPS Society.
Thomas Howard, external vice president of Pi Kappa Alpha, contacted Terri Klein, MPS Society
development director, and they had an opportunity to meet in person when the entire fraternity
showed up mid-stride through their event.
“It was a wonderful opportunity to meet with young
philanthropists who really care about the future of our
children,” said Terri. “Most of the young men had never
heard of MPS or a lysosomal disease and I answered a lot of
questions. This kind of acknowledgement and support brings
hope to our future fundraising for rare diseases. Fundraising
that can lead to additional treatments and a cure.”
After warming up and presenting a check for more than
$7,000, the group gathered back on their bikes to refresh with
some banquet food and ride back on the second leg of their
trip, 220 miles to South Carolina.
Do you have any FANs?
Of course you do, we all do; they help to keep us going. We call them:
Fundraising
Committee:
MaryEllen Pendleton, chair
Stephanie Bozarth
Jennifer Clarke
Ernie Dummann
Anne Gniazdowski
Tom Gniazdowski
Angela Guajardo
Steve Holland
Larry Kirch
Terri Klein
Dave Madsen
Hope Madsen
Laurie Turner
Barbara Wedehase
Family/Friends
And
Neighbors
FANs can be a large part of our support system and many are willing to do more than we ask of
them. They just don’t know how to help or what to do, and most of us don’t like to ask. Most of us
would do ANYTHING to help save our children, so why do we often feel guilty about asking for MPS
donations? Over the years, you probably have heard many people say, “I would love to donate to
MPS, and you should not feel guilty asking.” We challenge our doctors and schools daily as we fight
for the betterment of our childrens’ lives, so let us challenge ourselves to overcome the difficulties
of asking for financial support.
We are encouraging all of our MPS families and friends to ask your FANs for donations to help
support the National MPS Society’s mission: Support for our Families. Research for a Cure.
Here’s how to get started:
Developed by National MPS Society board members Tom and Anne Gniazdowski, FANs is a simple
fundraising program everyone can use.
1. Mail, e-mail or personally deliver a request note to your FANs (see sample, next page).
2. Envelope insert included with Courage.
3. Your FANs should return their donations to you.
4. Collect all of the donations and mail directly to the MPS office.
5. For additional envelopes, contact Terri Klein at [email protected].
That’s it. It’s easy, simple and rewarding. We hope you take this opportunity to help us help our MPS
children. Call or e-mail the MPS Society office for additional information or for any fundraising
assistance. For questions regarding FANs, send an e-mail to [email protected] or
[email protected].
continued
26
Fundraising News
Following is a sample request note that can be used to send to your FANs.
Dear friends and neighbors,
I am writing to you today to ask for your support in helping our family. As you may know, our
son/daughter has a rare genetic disorder called mucopolysaccharidoses or MPS. The occurrence
of MPS and related diseases is thought to be one in 25,000 births. Currently, there is no known
cure for these debilitating disorders; however, we are very encouraged by the amount and quality
of research that is being conducted around the world.
So…how can you help?
You can help by joining our family, along with the National MPS Society, in our efforts to
fight this disease by raising awareness to others and by making a financial contribution to help
continue the Society’s mission of funding research for a cure and providing support for our
families.
The money we raise will help families obtain durable medical goods such as hearing aids, special
wheelchairs, reinforced strollers, hospital beds and handicap accessible home improvements
which are not covered by insurance. It also will fund important research to find new treatments
and cures for all MPS and related genetic disorders.
Every donation received will make a difference in the life of a child. Please make your check
payable to National MPS Society and return it to me in the enclosed envelope. All donations
are tax deductible.
On behalf of our families, we thank you for your generosity.
Ways to GIVE
• Renew your membership or sponsor another family
• Gifts in honor of a special person
• Gifts in memory of a special person
• Matching gifts through your employer (check with your human resource office)
1. Request a matching gift form from your employer
2. Complete the employee section of the form
3. Mail to the Society and we’ll do the rest
• Contribute through the Combined Federal Campaign if you are employed by the federal government — CFC #10943
• Designate the Society as a member of your local United Way. You will need to supply them with the Society’s name,
address and Federal ID number (FEIN #11-2734849)
• Annual Fund donation
• Major gift (usually 10 times that of your Annual Fund gift)
• Planned gift
1. Bequest in your will
2. Charitable remainder trust or charitable gift annuity
3. Charitable lead trust
4. Life insurance policy
5. Gift of appreciated assets (stocks, mutual funds and bonds)
• Gifts may be applied to the Society’s general operating purposes or restricted to one of our designated programs.
CONTACT: [email protected] or 877.MPS.1001
27
Fundraising News
National MPS Society Receives 4-Star Charity Rating
“On behalf of Charity Navigator, I wish to congratulate the National MPS Society on achieving
our coveted 4-star rating for sound fiscal management. As the nonprofit sector continues to grow
at an unprecedented pace, savvy donors are demanding more accountability, transparency and
quantifiable results from the charities they choose to support with their hard-earned
dollars. In this competitive philanthropic marketplace, Charity Navigator, America’s
premier charity evaluator, highlights the fine work of efficient charities such as your
own, and provides donors with essential information needed to give them greater
confidence in the charitable choices they make.
“Based on the most recent financial information available, we have calculated a new
rating for your organization. We are proud to announce the National MPS Society has earned our
4-star rating for its ability to efficiently manage and grow its finances. Approximately a quarter of
the charities we evaluate have received our highest rating, indicating that the National MPS Society
executes its mission in a fiscally responsible way and outperforms most other charities in America.
This “exceptional” designation from Charity Navigator differentiates National MPS Society from its
peers and demonstrates to the public it is worthy of their trust.”
Ken Berger, President and Chief Executive Officer, Charity Navigator
Sponsor a Child for a Cure 2011—Don’t get left behind!
If your family has wanted to participate in a walk/run event to raise money for research we have created
that opportunity. This program reaches out to families and members of the Society that want to sponsor
an affected loved one or a child who has passed away, and help find cures for MPS and related diseases.
Walk/runs across the country will be participating in this program in 2011.
All you need to do is:
• Submit a photo of your loved one to the race you wish to participate
• Include the name of your child and address
• Get sponsorship and send to the National MPS Society
In turn, the event will:
• Assign a runner to participate on behalf of your loved one
• Send you a courage medallion and photo with your assigned participant
The assigned runners are inspired by our heroes of MPS. Together they pave the path of
continued hope. The photos and amount raised will be published in an upcoming Courage magazine.
For more information on the Sponsor a Child for a Cure program, contact Jennifer Clarke at
[email protected].
Fundraising Reminders
• Don’t forget to submit a brief article for Courage about your fundraising success stories and
suggestions—they are terrific resources for other families planning events.
• Check out the fundraising section on the Web site for more information or to post your
event.
• For free MPS Society brochures and donor envelopes, or to submit information for the Web
site or Courage, send an e-mail to Terri Klein at [email protected].
28
Keep in mind—the Annual 5K Walk/Run and the Annual Fund are great ways to raise money
for the National MPS Society.
Fundraising News | Walk/Run
11th Anniversary
Walk/Run Program
Raising Awareness and Funding Research
for MPS and Related Diseases
For 11 years National MPS Society members, families and friends have committed to walk/
run fundraisers throughout the country. Each of these events has brought us one step closer
to finding treatments and cures for MPS and related diseases. We continue to increase
awareness and funds for research by educating one person at a time.
This program continues to be a success year after year. Remarkably, these events have raised
almost $3.1 million since they began in 2000. In 2010, 13 walk/runs generated approximately
$250,000. Whether large or small, each event was successful at raising awareness, reaching
out among the community and involving thousands of people in cities nationwide.
The Society thanks all of the coordinators and many volunteers for their hard work at these
successful events.
11th Annual Run for Erin
Oct. 2, 2010
Event Coordinator: Stacy Peters
The Peters family along with 50
volunteers hosted the 11th Annual
Run for Erin. This year 268
participants ran, walked and raised
more than $19,000. The event is held
in honor of their daughter Erin, who
has MPS III. Since 2000, the event
has been held at Woodstock High
School, GA, and has raised more than
$220,000 for research. Erin has
attended this high school for four
years, and she rode in the pace car
with her father Tom Peters at the
start of the race. Volunteers helped
with the raffle which raised $1,600.
Run for Erin is held annually and
has a board of directors. Each year
the Peter’s family finds many rewards
in hosting the event as they are able
to combine it with a family reunion.
Family came from Kansas, Ohio and
Tennessee to participate.
and more than 175 participants.
The event was successful in many
ways. They raised more than their
original goal and had a number of
sponsors and friends from within
the community who came out to
support the Durcholz family and
MPS research.
Beat It for Benny
Sept. 25, 2010
Event Coordinators:
Annette and Emily Durcholz
BioMarin MPS
Run For Your Life
Sept. 26, 2010
Event Coordinator: Kathie Ward
Held in Terre Haute, IN, this was
the first event in honor of Benny
(MPS III). Emily Durcholz contacted
the Society and wanted to host an
event in honor of her brother Ben.
The Durcholz family raised almost
$15,000! They had 25 volunteers
BioMarin hosted its 8th annual
walk/run in Novato, CA. The event
included 180 participants and 25
volunteers with tremendous support
from corporate donors and families.
continued
29
Mackenzie’s Run for MPS
Oct. 9, 2010
Event Coordinators:
Steven and Jennifer Clarke
The weather was sunny and beautiful
and the event raised more than
$58,000, surpassing previous years!
Altogether supporters of this event
have raised more than $312,000 for
research. Additionally many new
families joined this year and BioMarin
participated in the Sponsor a Child for a
Cure program.
5 for FiVe, Memory Run Louis V
Sept. 26, 2010
Event Coordinator:
Michelle Storm-Butts
raising awareness for MPS. They had
a number of participants and many
volunteers as they snuck their event
in between the rain. This event raised
nearly $4,000. Shelly is thankful to
all the volunteers and looks forward
to another event in 2011!
Kassi’s Kause 5K Walk/Run
Aug. 28, 2010
Event Coordinators:
This walk/run was hosted in Saratoga
Springs, NY, in memory of Louis (L.B.)
Butts V who was diagnosed with MPS I
and who passed away on May 3, 2009.
The event hosted 135 participants
and 21 volunteers. The event was
inspiring for Michelle and her
family because many donations were
received, and the investment of time
from friends and the community
made it overwhelmingly successful.
They held a balloon release in
memory of L.B. at the beginning
of the race, a raffle and provided
awards for placed finishers. The
event raised more than $6,000 and
provided a platform for awareness
among the community about L.B.
and his heroic spirit with MPS.
Inspiring Ian’s Hope
May 15, 2010
Event Coordinator: Shelly Maddox
Friends and family gathered in
Amarillo, TX, to support Ian Adams,
(MPS III) and Inspiring Ian’s Hope
walk/run for research. The event
included a large purple pickup to
start the event, raffles, T-shirts, music,
food and a volleyball tournament.
This was a day filled with fun while
30
Trisha Offenbacker and Amy Adams
More than 300 participants in
Poquoson, VA, and surrounding
communities came out in support
of the 8th Annual Mackenzie’s 5K
Run/Walk for MPS.
The community has shown so much
support for this event over the
last eight years. It was wonderful
to see family, friends, high school
volunteers and Kiwanis members
come together to support the cause.
Four other MPS families and their
children attended this year. The
event raised $7,400 this year and
$137,000 since its inception.
Miles for MPS
Sept.12, 2010
Event Coordinator: Laurel Radius
Kassi’s Kause is a second-year
event held at Travis Air Force Base
in California. This event hosted
children’s activities, food vendors,
a silent auction and brought many
MPS families together from the
area. The event had more than 250
participants, 50 volunteers and raised
more than $7,500 for research.
The family spoke at the Society’s annual
conference about the importance of
raising awareness for MPS and their
appreciation for the community
support they receive for Kassi at our
annual conference.
Miles for MPS celebrated its ninth
annual walk/run in Grand Rapids,
MI. Seventy participants and seven
volunteers honored Chip and David
Radius (MPS II), raising awareness
and funds for MPS research.
continued
MPS Run For Their Lives
Nov. 20, 2010
Event Coordinators:
Scott Hardin and Steve Holland
this year coincided with the annual
conference at Knott’s Berry Farm.
Highlights of the event were
combining the event with the annual
family conference and establishing
the race course within the Knott’s
Berry Farm Theme Park. Snoopy
greeted participants at the front
gate. This event has raised more than
$340,000 in the past 10 years. There
are many families who form teams
and raise money for the event. They
include: Jack Pack, Team Scotty,
Team Browne and others!
Post Office Café Annual 5K
Run/1K Fun Run for MPS
Oct. 16, 2010
Event Coordinator: Kerri Rose
MPS Run for Their Lives was held in
Fort Worth, TX, for the ninth
consecutive year. This event is held
in honor of Maddie and Laynie
Holland and in memory of Spencer
Holland. There was a total of 271
participants who ran or walked and
30 volunteers who came out on a
mild sunny day to raise awareness
for MPS and funds for research. This
event raised approximately $9,500
in 2010 and more than 70,000
since the first event. Awards were
provided to many of the children
who participated.
MPS Walk/Run LA
Oct. 3, 2010
Event Coordinator: Tami Slawson
This third-year event held in
Guttenberg, IA, raised more than
$14,000 for research. The event is
held in honor of Ryan Hunt (MPS II).
They had 215 participants and 20
volunteers on an overcast day.
The event is supported by the
community and grows each and
every year. The Hunt family has
received donations from as far away
as Canada and we look forward to
watching their event grow.
Strides for Sara
June. 12, 2010
Event Coordinator: Monique Dickerson
Held in Babylon, NY, this event
raised more than $27,180 in honor
of Casey Lessing and in memory of
Mark Lessing Jr. for research.
The event had 530 participants and
60 volunteers on a sunny but cool
day in New York. This event has
grown within the community and
continues to raise awareness. The
race is a welcoming and professional
setting for the avid runner/walker.
The Post Office Café has raised more
than $100,000 in the past five years.
,
On a mild day in October friends
and families came out in support
of the 10th Annual MPS Walk/
Run LA. Four hundred participants
and many volunteers raised almost
$59,000. The annual event hosts MPS
families from around California;
River Run for Ryan
Aug. 14, 2010
Event Coordinators:
Jonathon and Marie Hunt
Miles for MPS participated in Sponsor
a Child for a Cure as an additional
outreach program for families. This
year the event raised more than
$4,000 and they have raised more
than $70,000 since their first event!
Hosting an event is rewarding
and helps everyone raise
awareness and critical funds for
research. For more information
contact Terri Klein, development
director, at [email protected].
The third annual Strides for Sara was
held in Fair Haven, NY, on a rainy day
in memory of Sara. There were 124
participants and 35 volunteers, but
despite the rain and low turnout the
community shined! The event raised
$3,900.
Monique said because of the rain,
sponsors and friends were bringing
checks to her family days after the
event had ended. The event included
a basket raffle and food. Despite the
road construction and other obstacles
the day was an absolute success! The
Dickerson’s are thankful for the
community support and look forward
to next year’s event.
31
Standing Ovation
Let’s Give a Standing Ovation To…
The Standing Ovation Award is intended to honor amazing people in our MPS
family for their resilience, courage, tenacity and passion for life while facing the
many challenges of having MPS. This award was created by Denise Dengel,
an adult with MPS I, who knows the daily struggles of living with MPS and
envisioned an award to honor the individuals who also battle MPS each and
every day.
We give a standing ovation to:
Sam Caswell, MPS I
I had a bone marrow transplant when I was 1 year old. My 6-year-old sister, Jenna,
gave me the gift of life for my first birthday. I have had 14 surgeries but I have
learned how to go through it with my family and friends. My family thinks I’m the
bravest kid in the whole world. They also think it’s funny that I get more excited
about going to Minnesota to see doctors and friends than going to Disney World!
I have always loved TV, movies and video games; right now my favorite TV show is
“How I Met Your Mother.” All my favorite movies have Adam Sandler in them and
it’s kind of funny because he is from the city right next to my town! My favorite
video game to play with friends is Mario Super Smash Brothers Brawl. I love reading
and am currently reading the Harry Potter series; my goal was to complete it by
July. I just started the seventh and last book, way ahead of schedule! I like to help out at Make-a-Wish
events and sometimes I give speeches and tell people it’s a great organization to give money to. So are
the MPS Society and the Ronald McDonald House. I do Special Olympics—basketball is my favorite
event. My top dribbling score is 298 in a minute! Winning the gold medal was great, but my proudest
accomplishments have been in school. I got a Citizenship Award at the end of eighth grade and made
up my mind I wanted to get all As in high school. The first two terms I did it! I got all A+s and As and
made the high honor roll. Another exciting thing about being in high school is that I get to ride my
segway in the halls because there is so much walking and it hurts my knees and legs. I think the most
important thing people can do is treat everyone the same.
Michael George, MPS II
Michael is a very active 7-year-old who loves being in first grade. He doesn’t talk
much, but he loves giving hugs and kisses. His favorite thing to do is eat; he may be
half boy/half bottomless pit. He enjoys watching Dora, Diego and Barney. Michael
likes being read to and is really getting into Harry Potter. He really loves being in
the water whether it is playing in the bathtub, lounging in the hot tub or swimming
in a pool. He loves spending time with his Grammy Kristin, Papaw Chuck and Papa
Dee. Michael’s biggest accomplishment this year is that he now walks to the bus all
by himself! My favorite thing about Michael is when he spontaneously bursts into
laughter—I would love to see what goes on inside his mind!
32
Standing Ovation
Nicholas Sprowles, MPS III
My name is Nicholas Sprowles and I am 17 years old. When I was 5 I was diagnosed
with MPS III. I live in Abingdon, VA. I have one sister, Jamie, who is 21 years old
and in college. She doesn’t have Sanfilippo, but she could be a carrier of the gene.
When I was younger I could run faster than anyone could catch me. I could run
up and down stairs without holding on. Now I cannot walk. I used to love to eat, as
a matter of fact, I would eat ANYTHING even if wouldn’t fit into my mouth. Now
I have a feeding tube.
The first thing I lost with this disorder was my sense of fear. I could not be left
alone for even a second. If there was danger I could find it fast. I slept very little
so this was a big job.
I have been an inspiration to everyone who has ever been in my life. Everyone
thinks I am an amazing little boy and is impressed with my strength and how
special I am. I have the best doctors in the world—my pediatrician here in
Abingdon and all of my doctors at the University of Virginia.
My smiles make the world go around!
Susan Quade, MPS IV
My name is Susan Quade and I am 34 years old. I have Morquio syndrome. I have
worked at the Charles County Sheriff’s Office in the Warrant Unit for more than
11 years and absolutely love it! I love spending time with my family and boyfriend.
I also love relaxing with my 15-year-old Yorkie. My biggest accomplishment so far
is buying my own place. My great uncle custom built my kitchen cabinets so I can
be fully independent and reach my counters without a stool. That was the most
rewarding experience. It was the first time in my life I was able to reach to turn
on water or see inside a pot on the stove without a stool. My parents were relieved
because now they know I am fully independent if something happens to them. I
also enjoy going to the beach and relaxing in the sun.
Isabel Bueso, MPS VI
Dorian Johnson, ML
From the moment Dori was born we knew he was born with a fighting spirit. He took us for surprise
when he was born with bleach-blond hair! He battled through weeks of tests, and weeks in the NICU
until we learned that Dorian and his twin brother, Wynn, were born with ML II. After getting over
the initial shock of what that diagnosis meant, we made a promise to Dorian and Wynn that we’d do
our best to give them the best life possible, no matter what.
In the past two years we have been amazed and often amused at his sense of humor and sense of
adventure. Whether it’s happily watching his 7-year-old twin brothers play, or scooting around on the
floor chasing Wynn, he laughs and has fun at just about anything he puts his mind to.
He loves all kinds of music and enjoys singing along to his favorite songs (“Wheels on the Bus” and
“Head, Shoulders, Knees and Toes”). No matter how upset he might be, if you say hi to him, he’ll
stop whatever he is doing to say hi back. His night nurse introduced him to YouTube so every night
he watches all his favorite nursery rhyme songs on her iPhone. He blows the best kisses, makes the
biggest parties out of bath time, and is always keeping his eye on his mama.
Dori has taught us all about patience, courage and unconditional love. We adore our little warrior
and are so proud of how he inspires everyone around him.
33
A Warm Welcome
34
A Warm Welcome
A Warm Welcome introduces new Society members/families and offers
members yet another chance to connect with one another. If you have a
moment, please contact the new family to say hello and welcome them into
our MPS family. If you have been a member for a longer period of time, but
would like to introduce your family to the rest of the Society, please e-mail
Laurie Turner at [email protected].
The National MPS Society welcomes the Leiva family from North Carolina.
My name is Laurie Leiva. My husband, Michael, is a
field artillery officer in the U.S. Army. Michael is
originally from New Orleans, and I am from Houston,
TX. Mike’s career in the army has taken us all over the
United States, but right now we’re enjoying the mild
weather in North Carolina. We have two children: our
daughter Olivia is 4 1/2 and has MPS III, and our son
Peter is 2. I worked in fundraising and development
prior to Olivia’s diagnosis but I am blessed to be able to
stay home with Olivia and Peter now.
Our path to diagnosis began in 2008. Olivia had
frequent ear infections and was having ear tubes put
in when the surgeon noticed a “genetic abnormality”
and suggested we follow up with a developmental
pediatrician. The genetic abnormality the ENT
surgeon noticed was a slightly bifid uvula which turns
out not to have anything to do with MPS, but we did
follow up with the developmental pediatrician. The
developmental pediatrician thoroughly examined
Olivia and gave her a clean, normal, bill of health. He
did recommend speech therapy and a follow-up in six
months. Olivia began receiving speech therapy and her
vocabulary grew.
When we returned
for the six month
follow-up Olivia
was
continuing
to meet all of her
developmental
milestones, and
even
exceeding
them in certain
areas. However,
the doctor noticed that her facial structure seemed to
have changed; he requested that I bring in pictures of
Olivia and he shared his concerns that she might have
a type of MPS. Initially the doctor stressed that he did
not think she had type III and we spent much more
time talking about some of the other types of MPS,
particularly those with enzyme replacement therapy as
a treatment option. I was almost six months pregnant
with our son Peter when we received the initial results
confirming that Olivia had MPS but we still didn’t
know what type. When we received the test results
confirming that Olivia had MPS III B we were shocked.
I was almost nine months pregnant and Mike was busy
completing training to prepare for a deployment to
Afghanistan. Olivia was 2 1/2 when she was diagnosed.
We left that appointment with a handful of brochures
from the MPS Society and a purple “courage” bracelet.
Peter was born and we were relieved to learn that
he was healthy and not affected. During late night
feedings with Peter I found myself researching
Sanfilippo syndrome online. Mike returned from a
month-long training mission to hear me announce
that we were going to North Carolina so that Olivia
could be evaluated by staff at UNC and the Program
for Neurodevelopmental Function in Rare Disorders.
I had read information about transplant and spoken
with doctors at Duke University. We decided to move
forward with transplant. Mike was able to be reassigned
to Ft. Bragg, NC, and we packed up and moved. Olivia
was admitted to Duke for her transplant on her 3rd
birthday in July of 2009. Olivia did well throughout the
transplant process but it was a rough couple of months
for us as a family. Peter was able to spend some quality
time with his grandparents at their ranch in Texas while
Olivia and I were inpatient at Duke. Mike did not have
to deploy to Afghanistan and was able to work during
the week at Ft. Bragg and relieve me on the weekends
at the hospital. Our family was finally reunited at the
end of 2009 as Olivia recovered from transplant.
Olivia is now 18 months post-transplant. She enjoys
playing outdoors and looking at books. Her favorite
movies are “Beauty and the Beast” and the “Princess
and the Frog.” Olivia started preschool in the fall of
2010 and she loves it! I have made thousands of copies
of those MPS brochures that I was given at the time
of Olivia’s diagnosis and Mike still wears his purple
“courage” bracelet every day. Moving cross-country
and the transplant process threw us for a loop and
I’m embarrassed that I let so much time slip by before
officially joining the MPS Society. We’re happy to be
here now. It took us several months to process Olivia’s
diagnosis, to understand how MPS has changed our
lives. We’re here now though, better late than never!
We look forward to participating in the Society that has
already given us so much.
If you would like to contact us, our e-mail address is
[email protected]. We also have a family blog
at www.mikelaurieandolivialeiva.blogspot.com.
Tara Matthews
15, MPS III, 2/26/11
25, MPS III, 10/28/10
William Cowin
Zachary Mullinax
18, MPS II, 12/31/10
13, MPS II, 1/1/11
David Downing
Cara Marie Sanford
16, MPS II, 5/27/10
17, MPS III, 12/27/10
Shalom Garcia
Peggy Shipley
36, MPS I, 1/13/11
53, MPS I, 10/30/10
Cameron Green
Joseph Titus
13, MPS II, 11/25/10
31, MPS II, 11/10/10
Grant Hickman
19, MPS III, 12/17/10
Cara Marie Sanford, MPS III
8/18/93–12/27/10
Cara Bara, our granddaughter, passed away Dec. 27, 2010. She fought the good fight. She got sick in
October and never really recovered. She managed to hang in there for her sister’s, dad’s and mom’s
birthdays. She also was with her family through the holidays, which meant so much to them. Her
uncles, aunties and cousins will miss her very much. We all have so many memories of all the great
times we had with her through the years.
We would like to thank the Montville, CT, school system, Oakdale Elementary, Mohegan Elementary,
and especially Tyl Middle School where she was a student in her later years. The staff, teachers,
nurses and her wonderful aides took such loving and great care of her. Bravo to Dr. Owen Ehrlich
who was with her for most of her life. You couldn’t ask for a better person and doctor.
Cara was probably one of the best-dressed girls at school, always color coordinated, right down to her
hair accessories. Every day when she came home her dad would give her a bath, not that she liked it
in her later years. Some children aren’t as fortunate as Cara was to have such great parents and sister.
There was nothing they wouldn’t do to make her quality of life the very best.
Remembering Our Children
Steffany Barnett
The song I sang to Cara was, “Cara the Bara with the meatball eyes, put ’em in the oven and you get
French fries!”
We love and miss you.
Poppy and NaNa (Anthony and Donna Cataldi Sr.)
35
February 2011
Legislative Update #54
Legislative
Committee:
Ernie Dummann, Chair
Debbie Dummann
Jeff Bardsley
Stephanie Bozarth
Chip Brady
Steve Holland
Austin Noll
MaryEllen Pendleton
Kim Whitecotton
Roger Chapin
Terri Klein
Kelly Rose
Laurie Turner
Barbara Wedehase
FDA Commissioner Meets with Patient Advocate Groups
U.S. Food and Drug Administration (FDA) Commissioner Margaret Hamburg, MD, met with
leaders and members of approximately 60 patient organizations at the National Organization for
Rare Disorders Washington, DC, office in January to share her vision for the FDA and hear patient
advocates’ hopes, needs and concerns. In a direct, freestyle dialogue, Dr. Hamburg responded to
questions on topics such as how the FDA compares benefits and risks of new products and how
it communicates its expectations to those developing products. MPS mom and board of directors
member Stephanie Bozarth spoke with Dr. Hamburg and left information with the commissioner,
including suggestions on how to benefit the review of new drugs and therapies which we believe can
make the FDA more responsive to our rare disease.
Rare Disease Caucus Update
In 2010 the Rare and Neglected Diseases Congressional Caucus was established to focus on bringing
greater congressional attention to the 7,000 known rare diseases that currently have no approved
therapy by working to ensure funding for research into these diseases, and providing a forum for
members of congress, families and advocacy organizations to exchange ideas and policy concerns.
Co-chairs include Rep. John Crowley (D-NY) and Rep. Leonhard Lance (R-NJ). Rep. Fed Upson (R-MI)
stepped down from his chair position due to Energy and Commerce Committee rules, however
he will continue to actively participate in the Caucus. On Feb. 1, Rep. Crowly and representatives
from several advocacy groups, including the Kakkis EveryLife Foundation, met with staff from
Rep. Upton, Lance and Crowley’s offices to discuss some general issues of concern regarding rare
disease drug development.
Federal Research Center Will Help Develop Medicines
The Obama administration has become so concerned about the slowing pace of new drugs coming
out of the pharmaceutical industry that officials have decided to start a billion-dollar government
drug development center to help create medicines. Creating a drug development center is a signature
effort of Dr. Francis S. Collins, director of the National Institutes of Health (NIH).
The new effort comes as many large drug makers, unable to find enough new drugs, are paring back
research. Promising discoveries in illnesses like depression and Parkinson’s that once would have
led to clinical trials are instead going unexplored because companies have neither the will nor the
resources to undertake the effort.
The initial financing of the government’s new drug center is relatively small compared with the
$45.8 billion that the industry estimates it invested in research in 2009. The cost of bringing a single
drug to market can exceed $1 billion, according to some estimates, and drug companies have typically
spent twice as much on marketing as on research, a business model that is increasingly suspect.
The NIH has traditionally focused on basic research, such as describing the structure of proteins,
leaving industry to create drugs using those compounds. But the drug industry’s research productivity
has been declining for 15 years, “and it certainly doesn’t show any signs of turning upward,” said
Dr. Collins.
The job of the new center, to be called the National Center for Advancing Translational Sciences,
is akin to that of a home seller who spruces up properties to attract buyers in a down market. In
this case the center will do as much research as it needs to do so that it can attract drug company
investment.
continued
36
Legislative Update | February 2011
This means that, in some cases, the center will use one of the institutes’ four new robotic screeners to
find chemicals that affect enzymes and might lead to the development of a drug or a cure. In other
cases, the center may need to not only discover the right chemicals but also perform animal tests to
ensure they are safe and even start human trials to see if they work. All of that has traditionally been
done by drug companies, not the government.
“None of this is intended to be competitive with the private sector,” Dr. Collins said. “The hope would
be that any project that reaches the point of commercial appeal would be moved out of the academic
support line and into the private sector.”
Whether the government can succeed where private industry has failed is uncertain, officials
acknowledge, but they say doing nothing is not an option. The health and human services secretary,
Kathleen Sebelius, sent a letter to Congress on Jan. 14 outlining the plan to open the new drug
center by October—an unusually rapid turnaround for an idea first released with little fanfare in
December.
Creating the center is a signature effort of Dr. Collins, who once directed the agency’s Human
Genome Project. Dr. Collins has been predicting for years that gene sequencing will lead to a vast
array of new treatments, but years of effort and tens of billions of dollars in financing by drug makers
in gene-related research has largely been a bust.
As a result, industry has become far less willing to follow the latest genetic advances with expensive
clinical trials. Rather than wait longer, Dr. Collins has decided that the government can start the
work itself.
“I am a little frustrated to see how many of the discoveries that do look as though they have therapeutic
implications are waiting for the pharmaceutical industry to follow through with them,” he said.
Dr. Collins’ ability to conceive and create such a center in a few short months would have
been impossible for most of his predecessors, who had nice offices but little power. But
congress in recent years has invested real budgetary and administrative authority in the
director’s office, and Dr. Collins is the first to fully use these new powers.
Under the plan, more than $700 million in research projects already under way at various
institutes and centers would be brought together at the new center. But officials hope that
the prospect of finding new drugs will lure congress into increasing the center’s financing
well beyond $1 billion.
Hopes of new money may be optimistic. Republicans in the House have promised to cut the
kind of discretionary domestic spending that supports the health institutes, and officials are
already bracing for significant cuts this year. But Dr. Collins has hinted that he is willing to
cannibalize other parts of the health institutes to bring more resources to the new center.
“There are some people that would say this is not the time to do something bold and
ambitious because the budget is so tight,” he said. “But we would be irresponsible not to
take advantage of scientific opportunity, even if it means tightening in other places.” For
the plan to go into effect by October, the administration must by law get rid of one of the
27 centers and institutes already in existence at the NIH—something that has never been
done before. So the administration plans to downgrade the National Center for Research
Resources, in part by giving some of its functions to the new drug center.
This article is an excerpt from The New York Times, written by Gardiner Harris, published
Jan. 22, 2011.
37
Research News: Hope for the Future
38
Surrogate Endpoint Trial (SET) for Individuals
with MPS III A
Sponsored by Shire Human Genetic Therapies
SET is a one-year study designed to study the natural progression of Sanfilippo A syndrome, or
MPS III A, in approximately 20 patients. The recent protocol amendment now allows for patients greater than
1 year of age to be eligible for participation.
During a period of 12 months participants in the study will be evaluated to assess the severity and
progression of MPS III A, as measured by developmental age and milestones, central nervous system
function (including cognition, speech and motor skills) and biochemical markers of the condition
(levels of heparan sulfate and its breakdown products in blood urine and cerebrospinal fluid).
Additional information can be found at www.clinicaltrials.gov (identifier NCT01047306), or contact:
Shire HGT Medical Information, 484.595.8850, [email protected] or Amy K. Fisher, MS,
CGC, Shire HGT Global Medical Affairs, at 857.413.9553 or [email protected].
MPS IV A Clinical Assessment Program (MorCAP)
The MorCAP has been designed to provide a better clinical understanding of the natural history of
MPS IV A (Morquio A) syndrome by measuring various aspects of the disorder, including endurance
and respiratory function in affected patients. These insights may help BioMarin Pharmaceutical Inc.
design future clinical studies. Participation in the MorCAP study will require one or more visits yearly
to a clinic or hospital for up to 10 years. Experimental drug will not be administered during these visits.
MorCAP is a multinational study and includes sites located in the United States, United Kingdom,
South America, Canada, Europe and Asia. For more information, call toll-free 1.866.961.8212.
Clinical Trials
MPS I
The Los Angeles Biomedical Research Institute at
Harbor-UCLA Medical Center in Torrance, CA,
and the University of Minnesota are collaborating
on two important studies of intrathecal enzyme
replacement therapy (ERT) for patients with
MPS I. A brief description of each study and
eligibility criteria are listed below.
A Study of Intrathecal ERT for
Spinal Cord Compression in Patients
with MPS I
The purpose of this research study is to find out
whether giving ERT (with Aldurazyme®) as an
injection directly into the spinal canal (called
intrathecal injection) can help reduce spinal
cord compression due to MPS I and can provide
an alternative to surgery.
Study participants will have:
• up to 16 intrathecal ERT treatments given one
to three months apart over one-and-a-half years
• physical examinations (general and neurologic)
• other diagnostic tests
• reimbursement/payment of travel expenses
A Study of Intrathecal ERT for
Cognitive Decline in Patients with
MPS I
The purpose of this research study is to find
out whether giving ERT with Aldurazyme® as
an injection directly into the cerebral spinal
fluid (the fluid around the spinal cord and the
brain) can stabilize (keep from getting worse)
or improve cognitive decline in patients who
have MPS I. The term “cognitive decline” refers
to a change for the worse in our ability to think
and learn. Difficulty with thinking, memory,
language, concentration and decision making
are some signs of cognitive decline.
continued
• up to 10 treatments given one to three months
apart over two years (treatment group) or four
treatments given three months apart beginning
at month 12 (control group)
• physical examinations (general and neurologic)
• neuropsychological testing for cognitive decline
and MRI of the brain
• reimbursement/payment of travel expenses
Additional information can be obtained at
www.clinicaltrials.gov; search under “mucopolysaccharidosis.”
Study centers other than at Harbor-UCLA may be
available. For more information, contact:
Patricia Dickson, MD
310.781.1399
Fax: 310.782.2999
[email protected]
Assistant Professor
MPS Research Laboratory
Division of Medical Genetics
1124 W. Carson St., HH1
Torrance, CA 90502
Los Angeles Biomedical Research Institute at
Harbor-UCLA Medical Center
Agnes Chen, MD
310.222.4160
Fax: 310.320.2271
[email protected]
Assistant Clinical Professor
Division of Pediatric Neurology
Box 468
1000 West Carson St.
Torrance, CA 90502
Los Angeles Biomedical Research Institute at
Harbor-UCLA Medical Center
MPS I Intrathecal ERT for
Children Being Considered for
Transplantation
The University of Minnesota has recently
obtained FDA approval for the delivery of
laronidase into the spinal fluid of children with
Hurler syndrome being considered for marrow/
cord blood transplantation. The goal of these
studies is to decrease the neuropsychologic
decline that has been observed in children with
MPS I from the time the patients are initially
evaluated to the time they are one year from
transplantation. The hypothesis is that there is a
significant delay in achieving sufficient enzyme
levels in the brain following transplantation, and
that this may be overcome by giving enzyme into
the spinal fluid until this occurs. Patients with
MPS I who are between 8 and 36 months of age
who have not previously received enzyme therapy
and are being considered for transplantation
at the University of Minnesota are eligible.
Patients receiving laronidase in the spinal fluid
also will be on intravenous laronidase prior to
transplant. The study will involve four doses
of laronidase given during a lumbar puncture
(spinal tap) approximately three months before
transplantation, at the time of admission to the
hospital for the transplant, three months after
the transplant and six months after the date of
the transplant. The principal investigator of the
study is Dr. Paul Orchard, who can be reached at
612.626.2961 or by e-mail at [email protected].
Alternatively, Teresa Kivisto, nurse coordinator
with the study, can be reached at 612.273.2924 or
[email protected].
Study participants will have:
MPS II
MPS II Intrathecal Enzyme
Replacement Clinical Trial
Shire Human Genetic Therapies is sponsoring a
clinical trial at the University of North Carolina
at Chapel Hill to learn if direct administration
of recombinant enzyme into the fluid around
the brain and spinal cord is safe and a possible
treatment for children with MPS II with
developmental delays. The principal investigator
for the clinical trial, “A phase I/II safety and
ascending dose ranging study of idursulfase
administration via an intrathecal drug delivery
device in pediatric patients with MPS II who
demonstrate evidence of central nervous system
involvement and who are receiving treatment
with Elaprase®,” is Joseph Muenzer, MD, PhD.
Currently there is no approved therapy for
treating the brain and spinal cord in patients
with the severe form of MPS II. The goal of this
study is to give a new preparation of iduronate2-sulfatase (idursulfase-IT) directly into the
fluid surrounding the brain and spinal cord
(intrathecal administration). The new form of
iduronate-2-sulfatase has not been used before
in patients with MPS II and is considered
investigational. It has not been approved by the
FDA or any other regulatory agency.
This phase I/II clinical trial is planning to enroll
16 patients with MPS II between the ages of 3
to 8 years with evidence of early neurocognitive
decline using an open-label, three-dose trial
continued
39
design. This clinical trial will initially have both
a treatment group (12 study patients) and a
control group (four study patients) with the
control group eligible to receive intrathecal
enzyme after a six-month observational period.
The monthly intrathecal administration of
idursulfase-IT will be given using a Port-A-Cath®
II Low Profile™ intrathecal implantable access
system manufactured by Smiths Medical MD, Inc.
that requires surgical implantation.
To be eligible for the investigational intrathecal
enzyme replacement clinical trial, study patients
need to have some developmental delay but
cannot be severely impaired, have received and
tolerated a minimum of six months of weekly
intravenous Elaprase® and have adequate hearing
(with or without hearing aids) to complete
developmental assessments. Patients with MPS II
are not eligible if they have a shunt for the
treatment of hydrocephalus, have had a cord
blood or bone marrow transplant, or have other
medical conditions that may place the individual
at an increased risk during the investigational
clinical trial.
For more information about the clinical trial,
contact Dr. Joseph Muenzer at 919.966.1447, or
the study coordinator, Heather Preiss, RN, at
919.843.5731 at the University of North Carolina
at Chapel Hill, NC.
MPS III
A Phase I/II Safety, Tolerability,
Ascending Dose and Dose Frequency
Study of Recombinant Human
Heparan N-Sulfatase (rhHNS)
Intrathecal Administration Via an
Intrathecal Drug Delivery Device in
Patients with MPS III A
Shire Human Genetic Therapies is developing a
sulfamidase enzyme replacement therapy (ERT)
for patients with MPS III A. rhHNS is being
administered into the cerebrospinal fluid via a
surgically implanted intrathecal drug delivery
device (IDDD), because when administered
intravenously it does not cross the blood brain
barrier.
This study is a multi-center, multiple-dose, dose
escalation study designed to evaluate the safety,
tolerability and clinical activity of up to three
dose levels (two doses [10 and 45mg] monthly
and one dose [45mg] every other week for six
months) of rhHNS administered via an IDDD in
15 patients with MPS III A ages greater than or
equal to 3 years of age.
40
The study is expected to be completed March
2012; the duration of the study for each patient
is nine months.
Patients who have completed all study
requirements in this study will be invited to
participate in an open-label extension study that
will be designed to evaluate long-term safety and
clinical outcomes of intrathecal administration of
rhHNS.
The phase I/II clinical study is being conducted
at two sites: Emma Children’s Hospital,
Academic Medical Center in The Netherlands by
Dr. Frits Wijberg; and the St. Mary’s Hospital
in Manchester, UK, under the direction of
Drs. Simon Jones and Ed Wraith. The letter to
the Society from Shire about the study can be
accessed on our Web site under the Clinical Trials
section.
Additional information about the clinical trial
can be obtained at http://clinicaltrials.gov/ct2/
show/NCT01155778?term=MPS+III+intrathe
cal&rank=1, or by contacting Tiffany Crump at
484.595.8257 or [email protected], or Daryll
Heron at +44 1256 894572 or [email protected].
MPS IV
BioMarin Pharmaceutical Inc. announced Feb. 1,
2011, that it has initiated a pivotal phase III trial
for N-acetylgalactosamine 6-sulfatase (GALNS
or BMN 110), intended for the treatment of
MPS IV A (Morquio A syndrome).
“In under two years, we have progressed the
GALNS program from Clinical Trial Application
to initiation of the phase III trial. We have received
FDA feedback and have finalized the design
of the phase III pivotal trial,” said Jean-Jacques
Bienaime, chief executive officer of BioMarin.
“The study will be conducted at approximately
40 centers worldwide including Brazil, Japan,
Taiwan, most Western European countries,
Canada and the United States. The trial is
expected to enroll approximately 160 subjects
and will be the largest ERT trial conducted.
There are no therapeutic options for MPS IV A
patients who have a high unmet medical need.
Initiation of this well-designed pivotal study is an
important milestone for both the company and
the MPS IV A community.”
The phase III trial is a randomized, double-blind,
placebo-controlled study to evaluate the efficacy
and safety of GALNS in patients with MPS IV A.
The study will explore doses of 2 mg/kg/week
and 2 mg/kg/every other week for a treatment
continued
Highlights from the phase I/II study:
• Endurance improvements with GALNS were
consistent with, and in some cases, better than
those observed in pivotal studies of approved
enzyme replacement therapies.
• Clinically meaningful improvements in two
measures of endurance (six-minute walk
distance and three-minute stair climb) were
achieved at both 24 weeks and 36 weeks as
compared to baseline.
• Clinically meaningful improvements in two
measures of pulmonary function (forced vital
capacity and maximum voluntary ventilation)
were achieved at 36 weeks as compared to
baseline.
• The frequency and severity of infusion reactions
were comparable to those observed with
Naglazyme® and Aldurazyme®.
period of 24 weeks. The primary endpoint is the
six-minute walk test, and the secondary endpoints
are the three-minute stair climb test and urine
keratan sulfate concentration.
Additional information can be found at
www.bmrn.com and http://clinicaltrials.gov/
ct2/showNCT00787995?term=MPS+IV&rank=1.
MPS VII
A gene therapy clinical trial for MPS VII, also
known as Sly syndrome, has been put on hold
pending additional data.
ML II/III
Currently there are no programs in place for
developing treatment options for ML II/III.
• Keratan sulfate levels decreased shortly after
the initiation of treatment and fell further as
the study progressed.
Treatment Therapies
MPS I
Aldurazyme®, administered once-weekly, has
been approved in the United States and in 15
countries of the European Union for long-term
enzyme replacement therapy (ERT) in patients
with a confirmed diagnosis of MPS I, to treat the
non-neurological manifestations of the disease.
Aldurazyme was developed by BioMarin and
Genzyme under a joint venture agreement that
assigns commercial manufacturing responsibilities
to BioMarin, and worldwide sales and marketing
responsibilities to Genzyme.
www.aldurazyme.com or by contacting Genzyme
at 800.745.4447.
MPS II
Elaprase® is a long-term ERT for patients with a
confirmed diagnosis of MPS II which has been
approved for use in the United States, Canada
and many countries in Europe. Elaprase was
developed and is produced by Shire Human
Genetic Therapies (formerly TKT), and is given
as weekly infusions to replace the missing enzyme
that Hunter syndrome patients fail to produce in
sufficient quantities.
www.shire.com or by contacting OnePathSM
toll-free at 866.888.0660. OnePath provides
assistance with insurance, product access,
treatment centers and education about Elaprase
and MPS II.
MPS VI
Naglazyme® is the ERT for individuals with a
confirmed diagnosis of MPS VI and has been
approved for use in the United States and
in many European countries. Developed and
produced by BioMarin Pharmaceutical, Inc.,
Naglazyme has been shown to improve walking
and stair-climbing capacity.
For more information, contact BioMarin Patient
and Physician Support at 866.906.6100 or
[email protected].
41
Resources | Helpful Information
Stop and Refuel Through Therapy
by Dr. Kimberly Frye
You may or may not know that I am a clinical
psychologist. Yet that is not why I am writing this.
This is a story about me and my family and how
I found it necessary to stop and refuel. There
was a time I thought I could do it all. Have a
successful career, a happy marriage, raise kids,
be a supportive friend, neighbor, you name it.
Failure was not an option, and anything less than
what I imagined was the best way to do things was
unacceptable.
Then one blurry day in June 2004 I learned I
had one more role to fill that I was completely
unprepared for: MPS parent. I was determined
to continue with my personal agenda, despite
the enormous adjustment my family was making.
Two months later my husband and I attended
our first MPS board meeting. A few months
after that we decided to run for the board and
found ourselves elected to our first term in
2005. I say “we” but it was more my idea than
his. I only mention this because it is just one of
hundreds of examples of compromise that have
resulted since our diagnosis. There would be
hundreds without MPS, but living with MPS just
increases that number in a way that can be hard
to recognize.
Since June 2004, the number of adjustments
we have made as a family and a married couple
The reason I decided to write this down is
because I believe therapy (support) is much
more than what I do for a living. It is what I do
to keep living. Support can come in many forms,
and tangible support for many MPS families just
does not come at all. Support when you think
you can do it all is almost impossible for anyone
else to give. Therapy is not the only way to help
yourself but it is the way I have helped myself
and my family. So I had to ask myself, “Why am I
not telling everybody?” Therapy is support tailormade for the person reaching out. I can hear it
already: How am I going to find the time? Where
am I going to find the money? Who is going to
watch my kids? Who is going to understand MPS?
Let me tell you a little story. I was driving one
evening with my 11-year-old, unaffected daughter.
We try to make a habit of one of us (my husband
or myself) taking her to dinner on infusion
day. This day it was my turn. I had spent the
day working, followed by errands necessary for
the Los Angeles conference, then preparations
for infusion and awaiting my husband’s return
from work so I could take my girl to dinner. I
Today my car may have run out of gas but it really means
that I have run out of gas. I must finally admit I cannot do it all…
have been great and, for the most part, healthy,
collaborative and ultimately successful. Or at
least that is what I used to think. The task of
trying to fill my predefined (or pre-diagnosis)
role of being a successful, functioning adult have
been put to the test over the last six years. I can
assure you that without the additional support
I have secured we certainly would not have the
healthy marriage or family I believe we have
been able to recreate and sustain. Without help I
know beyond a shadow of a doubt we would still
be married but we would be two people under
tremendous stress with less happy moments to
reflect on than painful, bitter and conflicted
ones filled with finger pointing, blame, sadness
and dissatisfaction.
Marriage is hard, parenting even harder, but
staying married and continuing to strive to
42
be a good (enough) parent living with MPS?
Well you already know of the challenge I am
speaking about. Almost impossible. Never
ending. Exhausting.
remember during the earlier part of the day
vaguely seeing my fuel light go on, but I was too
pressed for time to stop. Work until 2:30, pick up
Jack at school 2:40, fight with Jack about putting
EMLA on for “juice day;” pull out all infusion
supplies for nurse, home for 3:00 infusion; fight
with Jack about getting started…no you don’t
need to go to bathroom again, be gentle with
the dog; don’t touch your port after it’s been
cleaned; no we can’t play Wii now; yes we have
to do homework…sound familiar? Husband is
home and off we go. Lovely dinner, wonderful
catch-up conversation about school, friends,
life. Back in the car heading home, exiting the
freeway and that was it. On the exit ramp, with
a large blind spot curve behind me…we ran out
of gas! With my terrified daughter in the front
seat asking me with fear in her voice and eyes
what just happened. We were pushed to safety by
continued
If we do not take the time for ourselves, we all
will run out gas. When we deny the need to take
the time for ourselves, it will catch up us. It has
to. Therapy is my work, but as a patient it is my
time to focus on myself and get the support,
reassurance and feedback I need to recharge
and refuel myself and my marriage so we all
have the energy to keep going. It does not make
“it” go away, it does not “change” the reality of
all the stressors we face, it does not make what
we do as MPS parents, siblings or patients any
less difficult, but it does provide the space to
recharge, to connect, to express, to feel, to think
through, to breath. I have been on both sides of
the therapy chair and received as a patient what I
hope I provide as a doctor. That said, I think it is
important to consider how taking some time for
yourself could benefit you and your family, and
how not doing so can leave you with an empty
emotional tank on the side of the road one day
in the future.
Some tips for finding a therapist:
• Refer to your insurance plan and get a list of
mental health professionals. There are several
categories of mental health professionals to
consider:
– psychologists (PhD or PsyD)
– clinical social workers (LCSW or CSW)
–m
arriage and family therapists (MFT or
MFCC)
• Discard what these labels imply at first glance.
All of the above are licensed professionals
skilled in doing therapy. MFTs, LCSWs and
PhD/PsyDs all work with individuals and
families, so it’s not a concern if your insurance
will cover just one professional.
• The most important consideration when
looking for a therapist is finding a therapist who
is a good match with you, your personality and
how you think. I tell each and every new patient
that the first session is an opportunity for me
to get to know them AND for them to get to
know me. I let them know I will not ask them at
the end of the hour to “decide” if they want to
schedule another appointment but encourage
them to reflect on the time spent with me and
think about whether they felt heard by me
and comfortable speaking with me. If they
find they do and want to return we can make
an appointment; if they do not I will happily
help them find another potential therapist to
interview. Therapy does not work if you do not
feel heard or comfortable speaking with your
therapist. I always encourage potential patients
to meet with more than one therapist before
they decide. We don’t buy the first house we
see or the first car we drive and this goes for
therapy as well.
the highway patrol car but not before I utilized
every single anxiety reduction clinical skill in my
arsenal to keep my daughter (and myself) calm.
After calling my husband for help, who then had
to pull our son out of the bath, find a store to
purchase a gas can, purchase gas and then get to
us—it hit me. This is a warning sign that requires
my immediate attention. Today my car may have
run out of gas but it really means that I have run
out of gas. I must finally admit I cannot do it all,
and admitting that should not result in feelings
of shame.
• When you obtain your list of providers call at
least five. Listen to their message and trust your
reactions. I cannot begin to tell you how many
hang-ups I get on my voicemail. Over the years
I’ve learned that my patients call to listen to
my voice to get a sense of who I am before they
meet me face to face.
• Do not be surprised when some providers on
your list do not return your call. They have
actually done you a favor. If your therapist
cannot return your call when you are reaching
out for support what will they do when faced
with a crisis?
• Remember you are a consumer of a service.
If money is tight, discuss this at the first
appointment. You negotiate for a house and
a car and a therapist is no different. If you
can only pay $20 of your $40 co-pay, then say
this up front, and do not fear. Again, if they
are unwilling to negotiate, that is likely the
same person who will not understand when
you may need to cancel at the last minute
for something MPS related and charge you.
Speaking of this fact, bring it up first. The
reality is your life will present you with times
where you do need to cancel at the last minute
(in therapist terms less than 24 hours notice).
Inquire how this will be handled. We all make
exceptions and, yes, some rules are made to be
broken. I have an adolescent patient who has
survived three separate cancers. She has many
appointments and there are times when after
an appointment she does not feel well enough
to keep her appointment with me. She is one
where the 24 hour rule does not apply. Life is
life and again, rules are made to be broken.
We want our therapist to respect our reality,
the unpredictability that comes with MPS and
the challenges we can face when trying to plan
continued
Dr. Kimberly Frye
43
an appointment of any kind. Raising this issue
at the start of any therapeutic relationship
can put you at ease but it also communicates
to the therapist that this respect is mutual.
We still must call to cancel mind you, and as
soon as possible is best, but being aware of this
possibility at the start can reduce the level of
stress you experience.
• All therapists have an ethical and legal
obligation to their patients. All patients
have rights. Therapists cannot abandon us
or terminate treatment without a plan, and
they should not be discussing their problems
with you. As a therapist we all have different
styles. Some disclose more about themselves
than others do, but it should always be with a
clinical, supportive purpose. If you find you are
helping your therapist with a problem that is
not yours…run.
I hope this helps you see why it is so necessary to
stop and refuel. It is one way (and yes there are
many others) to ensure that you find the support
and balance we all need to keep going. It is much
easier to get where you want to go when you have
taken a moment to fill up your tank.
Social Security and Disability Resource Center
The Social Security and Disability Resource Center Web site (www.ssdrc.com) provides a detailed
overview of how the federal disability system works (Social Security disability and Supplemental
Security Income) and provides answers to many questions applicants typically have, but often have
trouble finding answers to. For the most part, the site is based on the author’s personal experience
as a former disability/Medicaid caseworker, and former disability examiner for the Social Security
administration.
Travel Medical Assistance
If you are going to a medical appointment in another state, following are some resources that may
be helpful regarding air travel. Some offer significant discounts or free travel.
American Airlines Miles for Kids (Need three weeks notice)
817.963.8118 www.aa.com/milesforkids
Angel Flight
978.794.6868 www.angelflightne.org
Miracle Flight for Kids (Need 15 days notice)
800.359.1711 www.miracleflights.org
National Patient Travel
800.296.1217 www.patienttravel.org
National Ticket Help
800.325.8908 North West Kids Cares (Need three weeks notice)
612.726.4206 www.nwa.com/corpinfo/aircares/about/kidcares.shtml
Operation Lift Off (Need seven to 10 days notice)
888.354.5757 www.operationliftoff.com
44
Lysosomal Storage Disease Clinical Care Network
Finding a doctor experienced in lysosomal storage disorders (LSDs) is often a challenge for patients.
A doctor to guide care and provide a place to receive infusions is what everyone deserves. That access
is critical to the best outcomes for LSDs, including MPS.
Dr. John Barranger has been acutely aware of the needs of many patients related to diagnosis,
monitoring, participation in the registry and a place to get infusions. Recognizing these unmet
needs, he has decided to focus his energy on providing expert care for LSD patients. Through a
new approach called the Lysosomal Storage Disease Clinical Care Network*, he seeks to provide
diagnostic and treatment services to patients in a site as close to their home as possible. At present,
he is determining where his clinical sites will be most useful for the LSD community.
Dr. Barranger will be the specialized doctor for the disease and responsible for the patient’s
case including their examination, collection of results and advice directly to each patient in the
office closest to their home. Availability is key to the sense of being well cared for. Access will be
provided 24/7.
Commitment to the development of the knowledge of LSDs is a serious responsibility, which he takes
as an obligation. Participation in the registry for each disorder will contribute important knowledge
of the disease and its response to treatment. At Lysosomal Storage Disease Clinical Care Network
sites, contribution of data to the registry will be done and the outcomes of registry studies shared
with patients.
In order to decide where to locate clinics to best serve the community, please let Dr. Barranger know
where you are and what services you need. Contact him at 412.735.5462 or [email protected]. * The Lysosomal Storage Disease Clinical Care Network is not affiliated with the LSD Research Consortium; it is not endorsed by the
National MPS Society.
First Hand Foundation strives to change children’s
lives around the world, one child at a time
In 1995, Cerner Corporation, a leading supplier of healthcare information technology, founded the
First Hand Foundation. A nonprofit organization, First Hand assists individual children with healthrelated needs when insurance and other financial resources have been exhausted.
The Foundation helps with clinical necessities (such as medication and surgeries), medical equipment
and travel related to a child’s care. Additionally, the Foundation implores doctors, hospitals and
equipment providers to discount their services below listed prices. Funding is distributed directly to
the child’s immediate healthcare provider.
Since inception, the First Hand Foundation has distributed $10 million to 85,000 individuals in
66 countries.
For more information or to apply for funding, go to www.cerner.com/firsthand.
Twist ‘n Write Penagain Pencil
makes writing easier for kids
TM
The Twist ‘n Write pencil is a new model specifically designed to fit smaller hands and bring back the
fun in writing. A thick 2 mm pencil lead never needs sharpening. The wishbone shaped design forces
the school-taught “tripod” position. It is great for those with special needs (carpal tunnel, arthritis,
ADHD and many more). It is being sold at Staples, Walgreens, Office Depot, CVS, Office Max and
Amazon for less than $2.00. For more information, visit www.penagain.com/twistnwrite.html.
45
Hunter disease e-clinic
Hunter disease e-clinic is a virtual training clinic, designed specifically for the purpose of learning to
diagnose and manage patients presenting with characteristic of rare genetic metabolic diseases. For
more information, visit www.sickkids.ca/lysosomalresearchgroup.
The Miracle League
The Miracle League is an organization that affords children with disabilities the opportunity to play
baseball. Players are paired up with a buddy and everyone has a chance to play—batting, running the
bases, etc. Even children without the use of their arms or legs can participate. The Miracle League
has more than 200 leagues across the country, as well as Puerto Rico, with 100 specialized rubberized
fields (for safety) and 100 fields under construction. It serves more than 80,000 children and young
adults with various disabilities.
For more information, visit www.miracleleague.com, call 770.760.1933, or send an e-mail to
[email protected].
Mini-Miracles
Mini-Miracles is an adaptive clothing line designed for the special needs child. Their philosophy is
to promote dignity, comfort and quality of life for every child, while giving back to the community by
donating to local Canadian charities.
Mini-Miracles clothing line consists of items chosen to meet the basic needs of the special needs
child. They reflect the research and experience that the owner, pediatric special needs nurse Victoria
Allen, has acquired working with special needs children.
The clothing is made of the highest quality Peruvian cotton and has different options available for
different needs (e.g., gastro-intestinal feeding tube access pocket, cut to fit diapers, etc.).
Featured adaptations:
• Flat seams to reduce friction
• Discrete adaptations so clothing looks normal
• Easy access—snaps, Velcro, stretchy fabrics
• Roomy seat to accommodate diapers (all ages)
• Longer rise in the back to accommodate
sitting in a wheelchair
• Elastic waist for ease of dressing and
increased comfort
• Meets Health Canada regulations for safety
and flammability
Suitable for:
• Limited range of motion and impaired
dexterity
• G-tube feeding
• Wheelchair
• Cerebral Palsy
• Muscular Dystrophy
• Developmental disabilities
• Contractures
• Toilet/changing needs
• Catheters and colostomy bags
• IV tubes
• Rigidity
Visit www.minimiracles.ca for more information.
Mercy Flight Southeast offers free flights
for those in need
46
The mission of Mercy Flight Southeast is “Coordinating free air transportation for children and adults
with medical or compelling humanitarian needs.” For more than 25 years, Mercy Flight Southeast has
coordinated these FREE missions, which are generously flown by a team of volunteer pilots who give
of their aircraft, their skills and their fuel, to help those who are in need. For more information, visit
www.MercyFlightSE.org.
Take Advantage of Family Support Programs
Please remember the Family Support Committee Program is here to help you, our members, with the
following programs:
• Medical Travel Assistance Program for medical appointments more than 200 miles from your home.
Grants help with transportation costs up to $500.
• Waived membership dues during times of financial difficulties.
• Family Assistance Program grants to help fund durable medical goods up to $3,000.
• $1,000 Continuing Education Scholarships help fund post high school education for affected
individuals, their parents, siblings, spouses and children.
• Conference Travel Scholarships of up to $1,000 help cover costs so your family can attend the annual
family conference.
• Extraordinary Experiences offers grants up to $1,000 to assist affected individuals have an extraordinary
experience, including school trips, educational conferences, special items, travel, etc. Check to see if
your idea can be covered too!
• Social Gathering Program funds up to $750 in costs to help you host a regional social event in your
area. Make time to renew old friendships and make new ones locally.
Drug Safety Information
www.fda.gov/cder/drugSafety.htm
The U.S. Food and Drug Administration (FDA) has added a new feature to its Web site that provides
consumers with specific safety information about individual drug products. The new Drug Specific
Information Web page presents information on more than 200 approved drugs. Each drug has a
“core page” with links to all information about the product on the FDA’s Web site, including general
risk information and any past alerts issued about the drug.
Health Care Coverage Options
www.nahu.org/consumer/healthcare
The National Association of Health Underwriters has created a database that provides information
on health insurance options for low-income U.S. residents and people who change jobs or have
pre-existing health problems. The Health Care Coverage Options Database provides a state-by-state
list of public health insurance programs for low-income residents, such as Medicaid and the State
Children’s Health Insurance Program. It also lists 32 states that offer high-risk health insurance pools
for those with pre-existing health problems.
Genetics Home Reference
http://ghr.nlm.nih.gov/handbook
This Web site contains a comprehensive “Guide to Understanding Genetic Conditions,” with basic
information about genetics in clear language and links to online resources.
47
Tissue bank aims to improve treatment
of developmental disorders
The National Institute of Child Health and Human Development (NICHD) Brain and Tissue
Bank for Developmental Disorder at the University of Maryland in Baltimore is a tissue resource
center designed to further research aimed at improving the understanding, care and treatment of
developmental disorders.
The NICHD Brain and Tissue Bank serves as an intermediary between the research community and
people who wish to donate tissue for research upon the time of their death. The Bank safely stores
the tissue until qualified researchers request the tissue for research which has been approved by their
Institutional Review Board. Both people with developmental disorders and people free of disorders
are encouraged to register and donate tissue. Often times it is the comparison of the unaffected with
the affected which unlocks the medical mystery of a disorder.
The availability of tissue from donors with MPS and other storage diseases is especially limited. As
more tissue becomes available and more researchers dedicate their life’s work to this disorder, new
discoveries can lead to new treatments and, perhaps one day, to a cure. It is only through the study
of donated tissue that important answers will be found.
If you are interested in becoming a registered donor, or if you have questions regarding the donation
process, contact Melissa Davis, project coordinator, at 800.847.1539.
Morgan’s Wonderland:
Finally, a place where everyone can play!
Located in San Antonio, TX, Morgan’s Wonderland is the world’s first ultra accessible family fun park
designed specifically for children and adults with special needs, their family members, caregivers,
friends and the entire community. This 25-acre park is a unique oasis which, through the spirit of
inclusion, allows people of all abilities to play, learn and share life-changing experiences together, in
a fun and safe environment.
For more information, go to www.morganswonderland.com/index.html.
Brave Community offers customized information
about rare diseases
BraveCommunity.com provides a dynamic source of news, information and personal insights about
certain rare diseases for patients and their families, friends and healthcare professionals. The Web site
searches the Internet and posts Web content and news stories relevant to certain rare genetic diseases
to help you stay informed. BraveCommunity.com also provides disease summaries, information
about the clinical trial process and relevant publications. Users can sign up to personalize content
and share own stories.
Learn more at www.BraveCommunity.com.
48
Grottoes brings smiles to special needs children
The Grottoes of North America Humanitarian Foundation is a not-for-profit organization dedicated
to funding dental care for children with special needs. Headquartered in Gahanna, OH, the group
believes “that children with special needs should live life as fully and as independently as possible. We
are passionately committed to helping alleviate the suffering and improving children’s quality of life
by providing dental care for those who otherwise might go without, One Smile at a Time.”
The Foundation’s Dental Care for Children with Special Needs program, established in 1970,
helps cover the costs of dental treatment, including hospital and anesthesia costs when needed,
for children under age 18 with cerebral palsy, muscular dystrophy (and related neuromuscular
disorders), organ transplant recipients or those with mental retardation. In addition to providing
funding, the program’s volunteers can help to connect special needs children and their families with
trained dental professionals.
Last year, the Grottoes’ Humanitarian Foundation paid $895,000 to assist 1,428 special needs
children. The program does not place a limit on the amount of support per child, but there are
maximum fees for allowable dental procedures. The only stipulation is that patients not be enrolled
in Medicaid.
For more information about the Dental Care for Children with Special Needs program, contact the
Grottoes of North America Humanitarian Foundation, 430 Beecher Road, Gahanna, OH 43230; call
614.933.0711; e-mail [email protected]; or visit www.hfgrotto.org.
Resources Available to California MPS Families
presented by Dr. Kimberly Frye at a California family meeting
Regional Centers can provide respite care, day care reimbursement, behavioral services and
counseling. A medical waiver is necessary to be eligible for in-home supportive services.
Regional Centers
Contact Information
Coverage Area
East Los Angeles
Gloria Wong, director
1000 South Fremont
Alhambra, CA 91802
Telephone: 626.299.4700
Fax: 626.281.1163
Web site: www.elarc.org
The Eastern Los Angeles Regional
Center serves the health districts
of Alhambra, East Los Angeles,
Northeast, and Whittier within the
county of Los Angeles.
Diane Campbell Anand,
director
3303 Wilshire Blvd., Suite 700
Los Angeles, CA 90010
Telephone: 213.383.1300
Fax: 213.383.6526
Web site: www.lanterman.org
The Frank D. Lanterman Regional
Center serves the health districts
of Central, Glendale, HollywoodWilshire and Pasadena within the
county of Los Angeles.
Frank D.
Lanterman
For a complete listing
of California Regional
Centers, go to www.dds.
ca.gov/RC/RCList.cfm.
continued
49
Regional Centers
Contact Information
Coverage Area
Harbor Regional
Center
Patricia Del Monico, director
Del Amo Business Plaza
21231 Hawthorne Blvd.
Torrance, CA 90503
Telephone: 310.540.1711
Fax: 310.540.9538
Web site: www.harborrc.com
The Harbor Regional Center
serves the health districts of
Bellflower, Harbor, Long Beach
and Torrance within the city of
Los Angeles.
North Los Angeles
County Regional
Center
George Stevens, director
15400 Sherman Way, Suite 170
Van Nuys, CA 91406-4211
Telephone: 818.778.1900
Fax: 818.756.6140
Web site: www.nlacrc.org
The North Los Angeles County
Regional Center serves the
health districts of East Valley, San
Fernando, and West Valley within
the city of Los Angeles.
Regional Center
of Orange County
Larry Landauer, Executive
director
801 Civic Center Drive West,
Suite 100
Santa Ana, CA 92701-4206
Mailing Address: P.O. Box 22010
Santa Ana, CA 92702-2010
Telephone: 714.796.5100
Fax: 714.547.4365
Web site: www.rcocdd.com
The Regional Center of Orange
County serves Orange County.
San Gabriel/
Pomona Regional
Center
R. Keith Penman, director
761 Corporate Center Drive
Pomona, CA 91768
Telephone: 909.620.7722/24 hrs.
Web site: www.sgprc.org
The San Gabriel/Pomona
Regional Center serves the cities of
El Monte, Monrovia, Pomona, and
Glendora within the county of Los
Angeles.
Please submit
information about
resources in your state
to Laurie Turner at
[email protected].
California Children Services
California Children Services (CCS) is a statewide program for children under the age of 21 who
have physical disabilities and meet specified financial guidelines. CCS provides medically necessary
Assistive Technology services and equipment related to the child’s eligible condition, including
durable medical equipment, medical supplies, medical appliances and electronic communication
devices. To apply for CCS services contact your local county health department.
If your child has a special health problem that is covered by CCS, then CCS may pay for or help with:
• doctor visits and care, hospital stays, surgery, physical therapy and occupational therapy, tests,
X-rays, medical equipment, and medical supplies
• medical case management to help get special doctors and to refer you to other agencies, such as
public health nursing and regional centers
• medical Therapy Program, which provides physical therapy and/or occupational therapy in
public school
• physical therapy
• occupational therapy
• financial assistance
50
• counseling
continued
Santa Barbara
345 Camino del Remedio
Santa Barbara, CA 93110
805.681.5360 or 805.681.4763
Ventura
2240 East Gonzales Road, Suite 260
Oxnard, CA 93036-8210
805.981.5281 or 805.981.5280
For a complete list of CCS offices, go to www.dhcs.
ca.gov/services/ccs/Pages/CountyOffices.aspx.
Los Angeles
9320 Telstar Avenue, Suite 226
El Monte, CA 91731-2849
800.288.4584 or 800.924.1154
In addition, families caring for a child with a CCS eligible condition sometimes need other kinds of
help. For instance, counseling support services. Your child or someone in your family might want to
talk with a counselor or therapist about how your child’s condition is affecting your lives. Ask your
child’s Nurse Case Manager about counseling support services. Families find this helps more than
they thought it would. For more information, ask your nurse case manager or call the Social Work
Unit directly at 1.800.972.1707.
In-home Supportive Services (IHSS)
The IHSS Program will help pay for services provided to you so that you can remain safely in your
own home. To be eligible, you must be over 65 years of age, or disabled, or blind. Disabled children
are also eligible for IHSS. IHSS is considered an alternative to out-of-home care, such as nursing
homes or board and care facilities, and is available for a maximum of 283 hours per month (or
$2,688 per month).
The types of services which can be authorized through IHSS are housecleaning, meal preparation,
laundry, grocery shopping, personal care services (such as bowel and bladder care, bathing, grooming
and paramedical services), accompaniment to medical appointments, and protective supervision for
the mentally impaired.
You may be eligible for IHSS if you:
• are a current recipient of Supplemental Security Income/State Supplementary Payment (SSI/
SSP); or
• you meet all the eligibility criteria for SSI/SSP except that your income is in excess of the SSI/SSP
income levels; or
• you meet all the eligibility criteria for SSI/SSP, including income, but do not receive SSI/SSP; or
• you are a Medi-Cal recipient who meets SSI/SSP disability criteria.
Orange County In-Home Supportive Services
714.825.3000
Los Angeles In-Home Supportive Services
1.888.944.IHSS (4477)
Ventura County In-Home Supportive Services
805.654.3236
51
MPS Classifications
52
Mucopolysaccharidoses (MPS) and related diseases are genetic lysosomal
storage diseases caused by the body’s inability to produce specific enzymes.
Normally, the body uses enzymes to break down and recycle materials in
cells. In individuals with MPS and related diseases, the missing or insufficient
enzyme prevents the proper recycling process, resulting in the storage of
materials in virtually every cell of the body. As a result, cells do not perform
properly and may cause progressive damage throughout the body, including
the heart, bones, joints, respiratory system and central nervous system. While
the disease may not be apparent at birth, signs and symptoms develop with
age as more cells become damaged by the accumulation of cell materials.
Syndrome
Eponym
Enzyme Deficiency
MPS I
Hurler, Scheie,
Hurler-Scheie
a-L-Iduronidase
MPS II
Hunter
Iduronate sulfatase
MPS III A
Sanfilippo A
Heparan N-sulfatase
MPS III B
Sanfilippo B
a-N-Acetylglucosaminidase
MPS III C
Sanfilippo C
Acetyl CoA: a-glycosaminide
acetyltransferase
MPS III D
Sanfilippo D
N-Acetylglucosamine 6-sulfatase
MPS IV A
Morquio A
Galactose 6-sulfatase
MPS IV B
Morquio B
b Galactosidase
MPS VI
Maroteaux-Lamy
N-Acetylgalactosamine 4-sulfatase
(arylsulfatase B)
MPS VII
b-Glucuronidase
Sly
MPS IX
Hyaluronidase
ML II/III
N-acetylglucosamine-1phosphotransferase
I-Cell, Pseudo-Hurler
polydystrophy
Board of
DIRECTORS
Steve Holland, president
Amy Holland
1752 Hilltop Circle
Fort Worth, TX 76114
817.625.6999
[email protected]
[email protected]
MPS I H-S parents
Kim Frye, vice president
Stephen Frye
3625 E. Thousand Oaks Blvd.,
STE 217
Westlake Village, CA 91361
818.263.7420
[email protected]
[email protected]
MPS II parents
Tom Gniazdowski, treasurer
Anne Gniazdowski
315 Meadowview Court
Springboro, OH 45066
937.748.8809
[email protected]
[email protected]
MPS II parents
Austin Noll, secretary
3735 Redwood Circle
Palo Alto, CA 94306
650.521.0089
[email protected]
MPS III parent
Jeff Bardsley
1209 Daviswood Drive
McLean, VA 22102
703.547.7087
[email protected]
MPS II adult
Stephanie Bozarth
6106 Larstan Drive
Alexandria, VA 22312
703.256.1980
[email protected]
MPS IV parent
Jennifer Clarke
186 Odd Road
Poquoson, VA 23662
757.868.7569
[email protected]
MPS III parent
Angela and Luis Guajardo
1815 Post Oak Road
Edinburg, TX 78539
956.287.2887
[email protected]
MPS III parents
Kristine Klenke
7604 Sherry Creek Road
Worden, IL 62097
618.888.2204
[email protected]
MPS II parent
Hope and Dave Madsen
16610 North 11th Street
Lakeland, MN 55043
651.331.9625
[email protected]
[email protected]
MPS I parents
MaryEllen Pendleton
56 E. Vinedo Lane
Tempe, AZ 85284
480.831.2157
[email protected]
MPS III aunt
Kim Whitecotton
1413 Emigrant Way
Modesto, CA 95358
209.544.2708
[email protected]
MPS II parent
Gordon Wingate
16319 Jordyn Lake
Tomball, TX 77377
832.498.1724
[email protected]
MPS III parent
President Emerita
Marie Capobianco
Ernie Dummann
Steve Holland
Mary Majure Couture
Linda K. Shine
STAFF
Barbara Wedehase,
executive director
[email protected]
Terri Klein,
development director
[email protected]
Laurie Turner,
program director
[email protected]
Kelly Rose,
administrative assistant
[email protected]
SCIENTIFIC
ADVISORY BOARD
Alessandra D’Azzo, Ph.D.
Gideon Bach, Ph.D.
Lorne A. Clarke, M.D.
Robert Desnick, M.D., Ph.D.
Matthew Ellinwood, D.V.M., Ph.D.
Louis Elsas, M.D.
Mark Haskins, Ph.D., V.M.D.
John Hopwood, Ph.D.
Laird Jackson, M.D.
Joseph Muenzer, M.D., Ph.D.
Elizabeth Neufeld, Ph.D.
Beth A. Pletcher, M.D.
Mark Sands, Ph.D.
Edward Schuchman, Ph.D.
William Sly, M.D.
Charles H. Vite, D.V.M.
Steven Walkley, D.V.M., Ph.D.
David Wenger, Ph.D.
Chester Whitley, M.D., Ph.D.
John H. Wolfe, Ph.D.
Ed Wraith, M.D.
PO Box 14686
Durham, NC 27709-4686
NON-PROFIT ORG.
U.S. POSTAGE
PAID
CHAPEL HILL, NC
PERMIT #74

Board of Directors a Dynamic Component of Society Check out the

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