Summer 2010 - The Canadian MPS Society

Transcription

Highlights
in this issue:
National Family
Conference Speaker
Profiles—page 6
First Announcement: The
12th International MPS
Symposium to take place
in the Netherlands in
2012—page 12
Research for a Cure: Four
research grants funded by
the Society—page 18
Highlights from the 2010
MPS CUP—page 20
Giving more than beans at
Tacos for Trey—page 26
Summer 2010
Please join us for our
National Family
Conference at the
Nottawasaga Inn Resort
in Alliston,
Ontario!
July 23-25,
2010
InThis Issue
Executive Director/Chair Reports/Welcome
4
Conference Speaker Biographies
6
Donations
7
Memorials & Birthday Greetings
8
Family News
10
Advocacy
11
Upcoming Conferences and Events
12
National Family Conference Program
13
Treatments & Clinical Trials
14
Clinical Studies Open for Enrolment
16
Society-Funded Research Projects
18
2010 MPS Cup Highlights
20
2010 Tacos for Trey Highlights
26
Fundraising
29
Society Merchandise
32
Society Forms
33
Family Assistance Program Funding Application
34
Society Publications
35
Scotiabank Toronto Waterfront Marathon
36
THE CANADIAN SOCIETY FOR MUCOPOLYSACCHARIDE
& RELATED DISEASES INC.
PO Box 30034, RPO Parkgate
North Vancouver, BC V7H 2Y8
Tel (604) 924-5130/1-800-667-1846
Fax (604) 924-5131
[email protected]/www.mpssociety.ca
Charity #12903 0409 RR0001
The Canadian MPS Society’s Mission Statement:
The Canadian MPS Society is committed to:

Providing support to families affected with MPS and
related diseases.

Educating medical professionals and the general public about MPS and related diseases.

Raising funds for research so that one day there will
be cures for all types of MPS and related diseases.
What are MPS & Related Diseases?
Lysosomal storage diseases (LSDs) are rare, progressive genetic
disorders. Often, these diseases are inherited from parents
who have no idea they carry the responsible recessive genes.
People born with MPS and related lysosomal storage diseases
cannot produce certain enzymes necessary for breaking down
and recycling cells. Consequently, these cells store throughout the bodies of those with LSDs, in areas including their
hearts, respiratory systems, bones, joints, and central nervous
systems.
Babies may show no signs of disease, but as more cells become
damaged and storage increases, symptoms begin to appear.
For more detailed information about individual diseases,
treatments and research, please visit our website at
www.mpssociety.ca or order one of our disease-specific booklets from the order form on page 35.
Support for families.
Research for a cure.
Aerodynamically, the
Smiling faces on the front cover:
dragonfly shouldn’t be
Nicklas Harkins & Olympian Cammi Granato
able to fly, but the dragonfly doesn’t care, so it goes
on flying
anyway.
-Anonymous
Page 2
Do you have family news, photos,
fundraising news or an interesting
article to share with
the Connection’s readers?
Newsletter submission deadlines:
Please submit by email, if possible,
to [email protected] or
[email protected]
Fall Issue:
Spring Issue:
Summer Issue:
Winter Issue:
February 1st
May 1st
August 1st
November 1st
The Connection
Board of Directors and Medical Advisory Board
Executive Director:
Kirsten Harkins
MPS I parent
(604) 924-5130
[email protected]
Jill Ley, Executive Assistant
Mary Nelis, Quebec Representative Robin Casey
Bedford, QC
MPS I parent
(450) 248-7376
[email protected]
Joe Clarke
Bernie Geiss
North Vancouver, BC
Professional
(604) 924-3451
[email protected]
[email protected]
Todd Harkins
Board of Directors: Judy Byrne, Chair
Guelph, ON
MPS I parent
(519) 836-5949
[email protected]
Carrie Nimmo, Vice—Chair
Vancouver, BC
MPS I aunt
(604) 255-5456
[email protected]
Jean Linden, Secretary
Prince George, BC
MPS III parent
(250) 564-3698
[email protected]
Brenda MacLean, Treasurer
Professional
(604) 980-7556
[email protected]
Barbara Boland, Newfoundland
Representative
St. John’s, NFLD
MPS III parent
(709) 753-7874
[email protected]
M.D., M.S.C.
Alberta Children’s Hospital
Calgary, AB
North Vancouver, BC
MPS I parent
(604) 929-8969
[email protected]
Aubrey Hawton
Moonstone, ON
MPS III parent
(705) 835-5288
[email protected]
M.D., Ph.D.
Hospital for Sick Children
Toronto, ON
Mark Ludman
M.D., F.R.C.P.C., F.C.C.M.G.
IWK Health Centre
Halifax, NS
Serge Melancon
M.D.
Montreal Children’s Hospital
Montreal, QC
Cheryl Rockman-Greenberg
M.D., C.M., F.R.C.P.
Winnipeg Children’s Hospital
Winnipeg, MN
Randall Linton
Toronto, ON
MPS III parent
(416) 223-0078
[email protected]
Julian Raiman
Deb Purcell
Tony Rupar
North Vancouver, BC
MPS II Parent
(604) 980-0930
[email protected]
M.D.
CPRI
London, ON
M.D.
Hospital for Sick Children
Toronto, ON
Sylvia Stockler
Medical Advisory Board:
M.D.
BC Children’s Hospital
Vancouver, BC
Lorne Clarke, Chair
M.D,C.M.,F.R.C.P.C.,F.C.C.M.G.
University of British Columbia
Vancouver, BC
Accountant: Quantum Accounting, Vancouver, BC
Canadian MPS Society Bank Account: TD Canada Trust
Summer 2010
Page 3
Executive Director’s Report
Once again it’s summer. The long,
dark (and wet, if you live in Vancouver like I do!) days are over and
we’re energized by the abundance
of sunshine and light, and by the
unmistakable happiness evident in
those who are enjoying holidays
from school and work, and spending quality time with their families
and friends.
Our conferences are opportunities for us to connect
with our MPS “family” and I look forward to seeing many of
you this July at our National Family Conference.
Personally, I’ve received a lot of sustenance this
spring from the outpouring of support toward the Society
through our annual MPS CUP Fantasy Hockey Game. Although we’ve organized this event eight times, I am still overwhelmed every year by those who give so much of themselves
to help us make a difference.
This year was particularly special as the event took
place on International MPS Awareness Day and we were
blessed by the presence of Mark Dant, who together with his
family has made an enormous impact on the international
MPS community. Mark’s eloquent presentation provided
inspiration for everyone in attendance and we thank him
sincerely for making the trip to Vancouver to be with us.
Please view the MPS CUP feature beginning on page
20 and take a look at all of the faces of those who care deeply
about our children. We raised a approximately $96,000
though this year’s event and that money will help us continue
to provide support to affected families and research for a cure.
In addition to the MPS CUP held in Vancouver this
May, the inaugural Minnesota MPS CUP was held May 1st. It
was very exciting to see the MPS CUP branch out to south of
the border and we thank Hope and Dave Madsen for organizing a fabulous event that raised awareness of MPS and funds
for the National MPS Society. We’re all in this together!
If you live in the Toronto area, please consider getting involved with the 2011 MPS CUP Toronto committee –
with two successful events in Canada, we can do so much
more! And, the best part of being involved is the feeling of
camaraderie and good-will that comes from working toward a
worthy goal: a brighter future for those affected with MPS and
related lysosomal storage disorders.
In our next edition of the Connection, I’ll have
news from the 2010 International MPS Symposium, being
held in Adelaide. We are so fortunate to have such a brilliant
group of minds focused on the same goal as us, and I am
looking forward to continuing the work we’ve begun!
Kirsten Harkins
A Warm Welcome to our New Members:
Brian & Gloria Done
Jonathan & Marie Hunt
Jonathan & Marie are the parents of Ryan Hunt (MPS II)
Jennifer Gilhuis
Jennifer is the mother of Sarah, Rachel & Matthew (MPS IIIB)
Angie Lombardo
Angie is the mother of Matteo Spina (MPS I H)
Heather Steele
Tracey MacLeod
Dan & Christina Priest
Dan & Christina are the parents of Stephen (MPS IV B)
Above Right: Monika Nelis-Dupont (MPS I H) with
Matteo Spina (MPS I H)
Right: Matteo Spina (MPS I H) at the hospital with his
Mom & Dad receiving his first infusion
Page 4
The Connection
Chair’s Report
Greetings to all from the Board.
I hope you are enjoying the summer we never had last year (if you
are from Ontario that is!).
The Board has been
busy getting ready for our upcoming National Family Conference
at the Nottawasaga Inn in Alliston, Ontario. We are very excited
by our lineup of speakers and are
looking forward to seeing all of
our members.
We are also using this conference as an opportunity to videotape some of our members and our speakers.
We hope to get enough footage to make both an informational short film and a fundraising short film for our Society. Thanks to Randall for introducing us to his friend who
will be doing the filming and the production. Don’t be
alarmed if you find yourself being filmed!
If you live in the Toronto area, we have again registered as an official charity with the Scotiabank Toronto
Waterfront Marathon on September 26, 2010. In return
for donations or pledges of at least $50.00, we will provide
your registration fee and a t-shirt. You can do the full or
half marathon, or for us mere mortals, you can walk, stroll
or push a wheelchair for 5 km. Whatever your ability, there
is a distance for you.
Enjoy all the summer has to bring and I hope to
see you in Alliston!
Judy Fowler Byrne
$$ Do you know about the Canadian MPS
Society’s Family Assistance Program? $$
Grants of up to $1,500.00 are available to individuals or families affected by MPS or a related
disease, and can be used to offset emergency or
non-emergency costs associated with care and
management. Do you need a special piece of
adaptive equipment that your insurance will not
cover, or will only cover a portion of? Do you
need home renovations to accommodate a
wheelchair? View our FAP guidelines at
www.mpssociety.ca under “support” and call with
any questions. An application form is available
for download on the website or on page 34 of
the Connection.
We also have Family Assistance Program funds
specifically allocated for respite care.
If you need a break from caring for your
child(ren), we would like to help! Call for details.
When a child is diagnosed with a serious illness, the day-to-day joys of childhood take a
back seat to the rigours of treatment and hospitalization. For more than 20 years, the
Starlight Children's Foundation has been dedicated to improving the quality of life for
children with chronic and life-threatening illnesses and life-altering injuries by providing
entertainment, education, and family activities that help them cope with the pain, fear
and isolation of prolonged illness. We understand what families go through when a child is sick, and how important it is to
find relief from constant worry and isolation. Our programs have been proven to distract children from their pain, help
them better understand and manage their illnesses, and connect families with others facing similar challenges so that no one
feels alone.
Unlike any other charity, Starlight offers a comprehensive menu of outpatient, hospital-based, and WEB offerings
that enable us to provide on-going support for children and families—from diagnosis through the entire course of medical
treatment. Programs include:

GREAT ESCAPES—Family activities and outings in the Greater Vancouver area, Victoria, Calgary, Ottawa, Greater
Toronto area, and Montreal (Vancouver-area families received free tickets to the Olympics and Paralympics!!)

FUN CENTERS with WII—In-hospital entertainment units

HOSPITAL HAPPENINGS—In-hospital craft kits, ER admit kids, and special events

STARBRIGHT WORLD ONLINE COMMUNITY & EDUCATIONAL PROGRAMMING
Research studies have found that Starlight's programs help reduce children's pain while helping them to better deal with,
understand, and manage their illnesses. In addition, Starlight programs help the child, parents, and siblings experience less
depression, anxiety, pain and isolation, and bond as a family. For more information, please visit www.starlightcanada.org.
Summer 2010
Page 5
Conference Speaker Biographies
Dr. Lorne Clarke: Dr. Clarke for the past 20 years, has been a researcher and clinician in biochemical
genetics with a special interest in lysosomal storage diseases. His research group was among the original
groups to define mutations at the IDUA locus in mucopolysaccharidosis type I (MPS I) patients and was the
first to develop a murine model for MPS I. In addition to being a chief investigator of phase III clinical
trials for enzyme replacement therapy in MPS I, Prof. Clarke’s group is currently developing methods to
monitor disease progression and the identification of biomarkers of the mucopolysaccharidoses. His group
is also involved in the creation of and use of murine models of other lysosomal storage diseases, including
Gaucher diseases.
Dr. Julian Raiman: Dr. Raiman received his paediatric training in the UK, followed by a one year fellowship in Australia. His metabolic training was completed at The Hospital for Sick Children, Toronto as Departmental Fellow between 2002-2004 before being appointed as Consultant in Paediatric Metabolic Medicine at the Evelina Children’s Hospital, London, UK in July 2004. He rejoined the Division of Clinical
and Metabolic Genetics at Sick Kids as Clinician Specialist in February 2007. He is currently the lead clinician for the LSD patients within the hospital, is a Project Investigator collaborating on a variety of ongoing
trials including being the site investigator for the Longitudinal study of MPS I, II and VI, a multicentre
collaboration under the NIH Lysosomal Diseases Network. He also sits on the North American Board for
the Hunter Outcome survey and the Medical Advisory Board of The Canadian MPS Society.
Dr. Kendra Bjoraker: Dr. Bjoraker is a pediatric neuropsychologist at The Children’s Hospital-Denver
with specialized training and expertise in neurodegenerative diseases. She currently serves on the neurometabolic, neurogenetic, and neuromuscular clinic care teams. She is involved in multi-disciplinary research
projects investigating the factors that predict and contribute to health outcomes and quality-of-life in individuals with genetic and metabolic diseases and to determine what factors influence individuals with these
diseases in order to improve their quality of life, particularly for children with mucopolysaccharidoses, mucolipidoses, and leukodystrophies. Her primary clinical interests include evaluation of the neuropsychological sequelae of these diseases and syndromes with health care providers, parents, and educators.
Robin-Lee Norris: Ms. Norris practises corporate commercial, real estate and wills & estates, planning
law and is a senior partner in the Guelph office of Miller Thomson. She has significant experience appearing before municipal councils and other related tribunals. Robin-Lee is a highly skilled mediator who uses
this expertise to assist her clients in problem solving particularly in the areas of Family Business and Wills
& Estates. Robin-Lee is active in the Guelph community and sits on various boards and committees, including the Board of Trustees for the University of Guelph and the Grand River Conservation Foundation.
Dr. Durhane Wong-Reiger: Dr. Wong-Rieger is President and CEO of the Institute for Optimizing
Health Outcomes and the President of the Canadian Organization for Rare Disorders. She has served on
numerous health policy advisory committees and panels, including Project Coordinator for the Policy Dialogues for the Romanow Commission on the Future of Healthcare in Canada. From 1984 to 1999, Durhane was professor of psychology at the University of Windsor in Ontario, Canada. She is author of two
books and many articles. She is a frequent lecturer and conducts workshops and training for business organizations and community groups.
Thanks to all the speakers presenting at our conference. In
addition to the speakers above, the following will be present:
Justin Van Herrewegen, MPS VI
Matthew Santos, MPS IV B
Page 6
Alana Jackson MSW and Patricia Martinez, MA, MSW,
RSW, Social Workers
Division of Clinical and Metabolic Genetics
The Hospital for Sick Children
The Connection
Donations
John & Elda Bannon
Ronald & Elizabeth Perry
In Memory of Jennifer Bowser
Linda & Richard Huvenaars
Kim Ryan
Barry & Shirley Bowser
Kim Ryan
Peter & Angela Sherbanow
Little League BC Celebrity Golf Classic
Heather Steele
In Honour of Jen Dawson on
The Hydrecs Fund
Dr. Sylvia Stockler
Mother’s Day
United Way of Halifax Region
Biomarin Pharmaceutical Inc. (Canada)
Oscar & Malca Casiro
United Way Burnaby
Dr. Joe T.R. Clarke
BMO Employee Charitable Foundation
Malcolm & Kharis Collins
Iffat & Nadeem Amin
Catherine & Jim Fowler
Claudette & Rob Amirault
Ruth Goff
Ursula Bauerle
Len & Norma Linton
Angelle Bourdon
Elena Miller
Lori Ann Bradley
Mary Olson
Joan & Andy Byrne
Ronald & Elizabeth Perry
Dr. Robin Casey
Lynne McCrea
Claudette & Benoit Chartier
Peter & Angela Sherbanow
Dr. Joe T.R. Clarke
Shire Human Genetic Therapies
Malcolm & Kharis Collins
Dr. Sylvia Stockler
Dan & Mary Lou Derbecker
Jennifer & David Elder
Mike & Brenda Drabek
Raajpreet Sekhon
In Honour of John Colavacchia
Maria Di Ilio
Nat & Andrea Di Ilio
Robert & Lori Di Ilio
In Honour of
Emma Rose Grenon-LeMaitre
Henry Kelsey Senior Public School
Donations to the MPS IVB
Research Fund in memory of John
Bertram Thomas
Gordon & Janet Fortune-Woodworth
Cliff Adams
Catherine & Jim Fowler
Phillip & Lesly Barkley
Verica Gacic
Paul & Jennifer Gilhuis
Carole Grenon
Aubrey Hawton
Dean & Leah Haywood
MPS CUP Donations – page 22
MPS II Research/Tacos for Trey
Donations— page 27
Carmelo & Mirella Boeri
Stanley & Janice Bourne
Bridgeview Marine Ltd.
Broadleaf Logistics
Bryan Zellweger Ltd.
Perry Huneault
Francie Hunt
In Memory of Patrick Ellis
Nick & Sonia Lanese
Joan & Andy Byrne
R.M. & J. Buzza
Canada Scaffold Supply Co. Ltd.
Fredrick Child
Jean & Nels Linden
Angie Lombardo
In Memory of Sal Petrucelli
Tracey MacLeod
Robert & Lori Di Ilio
Cobblestone Properties Ltd
Maurice & Diana Creagh
Ian Dalgleish
Nadin Noujeim
Sylvie Ouimet
In Memory of Nathan Linden
Christiane Parent
Patricia Husberg
Rob & Lisa Patriquin
Judy Byrne
Summer 2010
Valter & Laura Bianchini
Delroc Industries Ltd.
Dick's Lumber
Chris D'Odorico
Page 7
Donations
Patti & Antonio D'Odorico
Gordon & Lois Neely
Tim Spangler
Dawn Kistritz
Mr. & Mrs. Zoltan Nemeth
Cari Stadel
Donal Laing
William New
Stuart Olson Constructors Inc.
Kaye Leaney
Robert & Yvonne Newmarch
Tall Crane Equipment Ltd.
Darryl & Rita Dyck
Northern Building Supply Ltd.
Roy & Carole Teather
Goldwood Industries Ltd.
Martin & Donna Oates
J.B. Thomas
Jeff Lea
Margaret Paris
Two Pillars Construction Ltd.
Karin & Michael Hubbard
Kenneth Parsons
Ventana Construction Corporation
Bus & Arlene Innes
Jose Payawal
Barry Walsh
Insta-Space Storage Ltd.
Mr. G.L. Peterson
Westman Steel Industries
Irly Distributors Ltd.
Don Poon
Allan White
Johnsen Archer LLP
Dan & Tina Priest
Wilkinson Steel and Metals
Heidi & Darcy Kernaghan
William & Karen Rossum
Louise Winterbottom
Dale & Jackie Lebans
L. & G. Rycroft
Yard-At-A-Time Concrete (1988) Ltd.
Glen Leblanc
Sandy's Furniture
M.E. Thomas Ltd.
Andy Lee
Serj & Rishi Sangara
Tom Lee
Israel Zev Shafran
Remembering our Children:
Jennifer Bowser: May 3, 1986 – July 3, 2002, MPS IIIB
Matthew Di Ilio: October 3, 1990 – August 19, 2006, MPS III
Kevin Haines: January 7, 1961 – August 11, 1979, MPS III
2010 Membership Renewals
Earlier this year, you received an email reminder to renew your membership for 2010 online at
www.mpssociety.ca.
If we do not have your email address, you received a hard copy form in January.
Regardless of the time of year we receive your renewal, membership is for one calendar year only.
Your 2010 membership will be valid through December 31, 2010. When you renew online, you will receive a
PDF income tax receipt, your membership will be updated automatically, and the Society will save on printing and
postage costs. When renewing online, please first login to your account, then renew your membership and update your profile so that we have your consent to include your contact information in our membership
directory and to reprint your child(ren)’s photos, birthdates and/or memorial dates.
Please note that your profile must be updated annually.
You can find a Membership Form on page 33 if you prefer renewing the ‘old-fashioned’ way!
Thank you for your continued support!
Page 88
The Connection
Birthday Wishes
Happy Birthday!
July
David Mackay – July 4, 1979 – Mannosidosis
Laurie Patry Pelletier – July 4, 1995 – MPS IV A
Sebastien Cairns – July 11, 2000 – MPS II
Natasha Kaweski – July 11, 1994 – MPS IV
Nathaniel Phaneuf – July 18, 2002 – MPS II
August
Shawn Pyl – August 15, 1997 – MPS III A
September
Emma Hardman — September 1, 2004 – MPS I H
Andrew Perry – September 3, 1977 – MPS II
Damien Kaweski – September 3, 1992 – MPS IV
Matteo Spina – September 6, 2009 – MPS I H
Rhoda Dickinson – September 10, 1992 — Fucosidosis
Evan Santos – September 10, 2005 – MPS I H
Reagan Knight – September 12, 1997 – MPS I H
Jordana Kilgour – September 26, 1997 – MPS I H
Zineddine Lahiouel – September 28, 1999 – MSP III
We cannot print your child’s name on our Birthday list without your written consent to do so. We must receive this consent every calendar
year. If you would like your child’s name to appear on the Birthday list, please indicate on the current year’s Society membership form. We
apologize if any names are inadvertently missed. Just give us a call and we will make sure your child’s name appears in the next edition of
The Connection. Happy Birthday!
Summer 2010
Page 9
Family News
Emma Hardman (MPS I H)
Hanover, Ontario
In December of 2009, our family had the pleasure of going to the
National MPS Society's family conference in Orlando, Florida. Our
family, along with my mom and aunt. We planned on going a few
days early so we could take Bailey and Emma to DisneyWorld and
some of the other parks. Bailey loved DisneyWorld, Emma not so
much, the crowds were a little too much for her. With financial
assistance from the Canadian MPS Society we were able to enjoy the
parks, spend time with our family and friends, and make memories
that will last a lifetime
Jeff and I were asked to participate in a panel discussion with some
reps from Genzyme. Six sets of parents with children with Hurler's
Emma Hardman (centre) with friends at the
that had bone marrow
2009
National Family Conference in Orlando,
transplants particiFlorida.
These MPS I H girls have all had bone
pated. It was interestmarrow transplants.
ing to hear the different stories from all the
parents on how their children did through their transplants, and how they are
doing today.
Once the conference began, we were busy attending information sessions, break
out sessions with all the parents of Hurler's children (post bmt/stem cell transplants). We really enjoyed listening
to all the other parents stories, how
much their children had accomplished, and the complicaEmma and friend Teaghan (MPS I H)
tions, many surgeries etc. We
learned after listening to all the parents and seeing the MPS I Hurler’s children/teens, that Emma is very unique in the fact that she does not talk (in English
anyways). Our son Bailey calls it Emlish (Emma's English).
We had a great time visiting with friends we had met through websites, many
emails and the MPS/ML forum online. It’s so nice to visit and be
around families who know first hand what its like to live with a child with MPS,
the struggles, the joys of every accomplishment, and the grief of dealing with a life
Bailey & friend Riley—super
threatening day in & day out.. We had a great time, learned a lot of interesting
siblings of Emma and Teaghan
information, and made
memories with our Bailey, Emma, my mom, aunt and our MPS
friends that we will remember forever!!!
Thanks so much for helping us make this trip more enjoyable for
all of us!!
Becky, Jeff, Bailey and Emma Hardman
Please send us photos and
updates for our newsletter!
Left: Monika Nelis-Dupont (MPS I H) celebrates her 11th
birthday with friends at the hospital
Page 10
The Connection
Advocacy
ERT Funding for MPS I
All individuals requiring Aldurazyme enzyme replacement
therapy in Canada are being treated. We do not anticipate
any interruptions in treatment for patients receiving
Aldurazyme.
ERT Funding for MPS II
Since Elaprase enzyme replacement therapy for MPS II was
not recommended for funding by the Common Drug Review’s (CDR’S) committee, it has been up to provincial, territorial, and federal funders to either follow that recommendation, or decide to fund regardless. Children in BC, Alberta,
and Quebec are now being treated (albeit in Quebec, the
funding is through private insurance), and Ontario’s new
Drugs for Rare Diseases program is funding patients in Ontario on a case-by-case basis. Funding is not available in Sas-
katchewan or New Brunswick, and we are working to address
that situation. Please consider participating in our electronic
letter-writing campaign: visit our website for more ways you
can help secure funding for ALL individuals with MPS II
needing treatment.
Parents of Canadian children affected with MPS II,
and adults with MPS II, should discuss Elaprase with their
children’s, or their own, physicians. Shire has established a
1-800# for families interested in exploring potential funding
options available through private insurance. For more information on Elaprase or the Shire Reimbursement Support
Program, please contact Kirsten at the Society’s head office.
And What about MPS VI?
A small number of Canadians are receiving ERT to treat their
MPS VI. Funding decisions for Naglazyme will continue to be
made on a case-by-case basis.
Please write a letter to the Saskatchewan Minister of Health, Don
McMorris, encouraging his Ministry to approve funding for MPS II
patients. Visit the advocacy page of our website for details. Thank you!
3rd Annual Action Day on Parliament Hill
MPs are listening to Rare Disease patients!
Even better...they are acting on what they have heard.
Join CORD for the 3rd Annual 'Action Day on Parliament Hill' on September 30, 2010. Transforming
commitment to action. Travel bursaries are available. For more info, visit www.raredisorders.ca
The Canadian MPS Society’s tribute
card program is a special way of
paying tribute to a friend or
a loved one.
On June 5, 2010 Kirsten Harkins (left) and Simon Ibell
(front row, left) attended the Canadian Gaucher Foundation’s Board Meeting in Toronto. We look forward to working with our friends in the lysosomal storage disorder community toward our common goals. Please do join us for
CORD’s 3rd Annual Action Day (see above) in Ottawa.
Summer 2010
Send an MPS tribute card for any occasion
as a meaningful gift to support children affected with MPS: to welcome a new baby, to
celebrate a birthday or anniversary, to memorialize the passing of a friend or relative or to
say thank you to someone special. MPS tribute cards may be ordered by making a contribution of $10.00 or more. Contact the office
with your request: sympathy or tribute cards
are sent out the same day and income tax
receipts are issued.
Page 11
Upcoming Conferences & Events
Upcoming conferences & events:
Invitation
July 23-25, 2010: The Canadian MPS Society’s 2010 National Family Conference.
Alliston, ON. Please join us! www,mpssociety.ca
August 27-28, 2010: Expert Meeting on Sanfilippo Disease (MPS III). Northampton,
England. www.mpssociety.co.uk
September 30, 2010: CORD’s Action Day on Parliament Hill. Ottawa, ON. Followed
by CORD’s Annual Conference on Rare Diseases. www.raredisorders.ca
February 16-18, 2011: 7th Annual WORLD Symposium, Las Vegas, Nevada.
www.lysosomalstoragenetwork.org
June 28-July 1, 2012: 12th International MPS Symposium, Noordwijkerhout, The
Netherlands. More information to follow in upcoming editions of the Connection.
The proposed theme of the 2012
International Symposium is:
“Patients, Doctors and Specialized
Networks for Optimal Care and
Innovative Treatment”.
At this symposium we aim to gather
all experts, including parents, patients, medical doctors, paramedics,
scientists and industry at symptom
specific plenary and disease specific
parallel sessions.
The programme will focus on subjects that underline the need for the
development of sustainable networks for expertise on MPS and
related diseases. Special attention
will be given to those disorders still
lacking curative treatment. Cross
cutting themes could be: orthopedic problems in MPS and neurosurgery in MPS.
Some of the topics that will be
covered at the family level are:
-Living with MPS: doctors, scientists
and industry as partners
-Special needs for the elder adult
patient with MPS
-Quality of life and continuous care
for patients with Sanfilippo and
Morquio disease
-New emerging therapies for MPSdiseases (including late-breaking
news)
Some of the topics that will be
covered at the professional level
are:
-Expectations regarding quality of
life research: do we need disease
specific scales?
-The importance of physician
driven registries and patient reported outcome surveys.
-New insights in tissue targeting and
treatment development
-(Long term) clinical effects of ERT,
HSCT & other emerging therapies.
We hope to welcome you in
Noordwijkerhout in June 2012!
Hanka Meutgeert,
Executive Director, VKS
Page 12
The Connection
2010 National Family Conference
THE CANADIAN MPS SOCIETY’S
NATIONAL FAMILY CONFERENCE
July 23 – July 25, 2010
Nottawasaga Inn Resort, Alliston, Ontario
Please join us at the beautiful Nottawasaga Inn (one hour from Toronto) to learn more about new research, treatments and
care strategies, and – perhaps most importantly – meet with other families, share experiences and create life-long friendships.
This year, we are pleased to offer presentations by our medical advisory board members Dr. Julian Raiman from The Hospital
for Sick Children in Toronto, and Dr. Lorne Clarke from BC Children’s Hospital. We are also very excited that Dr. Kendra
Bjoraker, a pediatric neuropsychologist with a long history of working with MPS patients—will be joining us for what promises
to be an incredibly educational and inspiring weekend.
Preliminary Schedule: (subject to change)
Friday, July 23, 2010
2:00 PM—6:00 PM
4:00 PM—7:00 PM
6:00 PM—8:00 PM
Saturday, July 24, 2010
7:30 AM—8:30 AM
8:30 AM—12:45 PM
8:30 AM—8:45 AM
8:45 AM—9:45 AM
9:45 AM—10:25 AM
10:25 AM—10:35 AM
10:35 AM—11:00 AM
11:00 AM—12:00 PM
12:00 PM—12:45 PM
1:00 PM—2:00 PM
2:00 PM—5:00 PM
2:00 PM—3:00 PM
2:00 PM—3:00 PM
3:00 PM—3:30 PM
3:30 PM—4:00 PM
4:00 PM—4:30 PM
4:30 PM—5:00 PM
6:00 PM—8:00 PM
Sunday, July 25, 2010
7:30 AM—8:30 AM
8:30 AM—11:30 AM
8:30 AM—9:30 AM
9:30 AM—10:00 AM
10:00 AM—10:30 AM
10:30 AM—11:30 AM
12:00 PM—1:30 PM
Optional golf outing (pre-registration required)
Registration
Welcome Reception/BBQ
Registration/Child and Youth Program Registration
Child/Youth Program
Welcome
Overview and Management—Dr. Lorne Clarke
Update on Clinical Studies & Importance of Registries—Dr. Julian Raiman
Panel Q & A—Dr. Clarke & Dr. Raiman
Coffee Break
Education & MPS—Dr. Kendra Bjoraker
Syndrome Breakout Groups
Lunch
Child/Youth Program
Adolescence and MPS—Dr. Kendra Bjoraker (concurrent session)
Independent Living for Adults with MPS (concurrent session)
Moving Toward Home Infusions—Dr. Julian Raiman
Financial Planning for families and individuals affected by rare diseases—
Robin-Lee Norris
Financial Resources available to families and individuals affected by MPS—
Alana Jackson & Patricia Martinez
Living Fully with MPS VI—Justin van Herrewegen
Dinner—everyone; followed by remembrance ceremony
Registration/Child and Youth Program Registration
Child/Youth Program
Research Updates—Dr. Lorne Clarke
Hope on the Horizon: New regulations & programs for Canadians with Rare
Disorders—Dr. Durhane Wong-Reiger
Living fully with MPS IV B—Matthew Santos
Canadian MPS Society Annual General Meeting
BBQ luncheon—everyone
Registration: Registration forms are available for download on our website.
Accommodations: Conference attendees must make hotel reservations by calling the Nottasawaga Inn Resort at (416) 3645068 and quoting the Canadian MPS Society. A group rate of $128.00 per night is available. We hope to see you there!
Summer 2010
Page 13
MPS I:
that has been observed in children with Hurler from the time
the patients are initially evaluated to the time they are one
Aldurazyme (laronidase) enzyme replacement therapy was
licensed for use by Health Canada on May 31, 2004, for long- year from transplantation. The study will involve four doses of
term treatment in patients with a confirmed diagnosis of MPS laronidase given during a lumbar puncture approximately
I, to treat the non-neurological manifestations of the disease. three months before transplantation, at the time of admission
to the hospital for the transplant, three months after the transFor more information on Aldurazyme, please visit
plant, and six months after the date of the transplant.
www.aldurazyme.com
Principal Investigator Dr. Paul Orchard can be contacted for
more information at 612-626-2961 or [email protected].
MPS I Intrathecal ERT for Spinal Cord Compression:
A one-year extension study has been approved. Enzyme replacement therapy (ERT) has been developed for MPS I. ERT MPS II:
helps many physical ailments due to the disease, but does not On June 15, 2007 Elaprase enzyme replacement therapy was
approved by Health Canada for the treatment of MPS II.
treat the central nervous system due to inability to cross the
blood brain barrier. The purpose of this study is to test delivShire MPS II Intrathecal Study:
ery of ERT to the spinal fluid via intrathecal injection in patients with MPS I. In this pilot study, recombinant human α- Dr. Joseph Muenzer will be conducting a Phase I/II
(Safety/Dosage) trial using intrathecal ERT for the treatment
L-iduronidase will be administered intrathecally once per
of MPS II, with a goal of preventing central nervous system
month for four months to individuals age 8 and older with
the Hurler-Scheie and Scheie forms of MPS I and spinal cord involvement. This study will take place at the University of
compression. If successful, intrathecal delivery could represent North Carolina in Chapel Hill, NC. If you are interested in
a practical, straightforward method of treating central nervous obtaining more information about the clinical trials, please
contact Dr. Muenzer (919)966-1447, or study coordinator
system disease due to lysosomal storage.
Heather Preiss at (919)843-5731 or [email protected].
Primary Outcomes:
Safety of intrathecal enzyme treatment by blood and spinal
fluid tests each month; improvement in neurologic signs reMPS III:
lated to spinal cord compression, by neurologic examination
Dr. Grzegorz Wegrzyn of Poland has been researching suband Japanese Orthopedic Association Scale each month; imstrate reduction therapy and the effect of genistein on syntheprovement in neurologic symptoms related to spinal cord
sis of glycosaminoglycans. A clinical trial will be beginning
compression, by subjective assessments and independence of
soon in the UK.
functioning scale each month; improvement in mobility, by
six-minute walk test each month; improvement in spinal cord Shire Pharmaceuticals Group, as part of its research to evaluate new approaches to the problem of treatment of the central
compression by MRI imaging and somatosensory evoked ponervous system, is hoping to move its MPS III-A program fortentials at baseline and four months; improvement in lyward. www.shire.com.
sosomal storage by spinal fluid glycosaminoglycan levels at
each treatment.
MPS IV:
Secondary Outcomes:
Improvement in spinal fluid pressure, by opening pressure
BioMarin Pharmaceutical Inc. announced April 21, 2009 the
measurements at each intrathecal treatment; improvement in initiation of a phase I/II clinical trial for BMN-110 or Nhydrocephalus and other brain lesions by MRI at baseline and acetylgalactosamine 6-sulfatase (GALNS), intended for the
four months.
treatment of MPS IV A.
Expected Total Enrollment: 10
Phase I/II clinical trials (safety) began in Q1 2009 with about
40 patients in the UK and there are about 20 enrolled in the
Additional information can be obtained at
http://www.clinicaltrials.gov/ct/show/NCT00215527?order= Phase II study. The Phase III trial will likely include 50-100
1 or by contacting Principal Investigator Dr. Patricia Dickson patients in several centres, and should begin sometime in late
2010.
at 310-222-4145 or [email protected].
The link to BioMarin’s MPS IV A program website is
MPS I Intrathecal ERT for Children Being Considered for www.morquiobmrn.com - this site is a great place to look for
Transplant:
current information. It will be updated at least four times per
The University of Minnesota in Minneapolis has recently ob- year, and of course, more often if there is significant news. In
tained FDA approval for the delivery of laronidase into the
addition, visitors have the option to register on the website to
spinal fluid of children with MPS I (Hurler syndrome) being
receive news about updates via email.
considered for marrow/cord blood transplantation. The goal
of these studies is to decrease the neurophsychologic decline
Page 14
The Connection
Further information about MorCAP is also available on ClinicalTrials.gov at this link;
http://www.clinicaltrials.gov/ct2/show/NCT00787995?term
=morquio&rank=2.
Patients or physicians who are interested in participating in
MorCAP (see announcement on page 20) can contact the
Montreal site’s study coordinator, Gail Ouellette at
[email protected] or phone: 819-543-0550 for
more information.
If you have particular questions about operational aspects of
BioMarin’s trials, you can contact Candice Henkel at
[email protected] or 415-506-6973.
For additional medical questions related to MPS IVA and the
clinical details of BioMarin’s program, please contact the
Medical Director for BioMarin’s MPS IVA program, Celeste
Decker, at [email protected] or 415-506-6469.
Pompe Disease
Myozyme® (alglucosidase alfa), a recombinant form of human acid alpha-glucosidase (GAA), was approved by Health
Canada on August 14, 2006 for the treatment of patients
with Pompe disease (GAA deficiency). For more information
on Pompe disease, please visit Canadian Association of
Pompe’s (CAP’s) website: www.pompecanada.com.
Alpha-Mannosidosis:
Study of outcomes from transplants for Alpha-Mannosidosis
Dr. Martin Myranek from Hanover Medical School is conducting a study on the outcomes of transplants for AlphaMannosidosis. ISMRD is encouraging all families to give
permission for the doctor who transplanted their child to
provide data to Dr. Myranek. The information obtained
should provide important knowledge about the efficacy of this
treatment. Copies of the covering letter and the data sheet
MPS VI:
for this study are posted on the Penguin Café at
The U.S. Food and Drug Administration (FDA) granted mar- www.mannosidosis.org.
keting approval for Naglazyme(TM) (galsulfase), the first specific therapy approved for the treatment of MPS VI , on June Clinical Trial of Enzyme Replacement Therapy for Alpha1, 2005 .
Mannosidosis
Editor’s note: There is no word on when (or if) BioMarin will apply The Scandinavian company Zymenex has been working for
for licensing approval for Naglazyme in Canada. For more informa- over 10 years to develop a number of Enzyme Replacement
tion, please contact BioMarin Patient and Physician Support (BPPS) Therapies for Lysosomal diseases, and have been closely inat 1-866-906-6100 or [email protected].
volved with the European consortium HUE MAN which was
funded by the European Commission. The HUE MAN consortium’s work involved studies about natural history of the
MPS VII:
disease, characteristics of the enzyme, and production methA gene therapy clinical trial for MPS VII, also known as Sly
ods and study of the effects in a mouse model of the disease.
syndrome, has been put on hold pending additional data.
ISMRD understands that the company hopes to commence a
Phase 1 clinical trial in Alpha-Mannosidosis patients some
time in 2010. We look forward to more news about this exciting development and we will pass it on to families as soon
as more is known. -from the ISMRD 2009 Winter newsletter
ARE YOU INTERESTED IN THE INTERNATIONAL MPS SYMPOSIUM
THAT JUST TOOK PLACE IN ADELAIDE? ALL PRESENTATIONS
WILL BE AVAILABLE ONLINE—DETAILS TO COME IN THE FALL
ISSUE OF THE CONNECTION OR CHECK OUR WEBSITE FOR A
LINK. REMEMBER, ALL THE SESSIONS FROM THE VANCOUVER SYMPOSIUM IN
2008 ARE STILL AVAILABLE AS WELL FOR VIEWING ONLINE!
All scientific sessions in Vancouver were videotaped and are available for viewing, along with their accompanying slideshows, at www.goldinfo.org. To view the sessions on the GOLD website, you will need
to register if you have not done so before. Registering only needs to be done once. Click on “Video Presentations” under “Education and Information” on the GOLD homepage’s left sidebar. Then, click on “register here”
in the first paragraph, which will open a registration form. You will be asked to give your email
address as a username, choose a password, and select the organisation of which you are a
member: Please select Canadian Society for Mucopolysaccharide & Related Diseases Inc. from
the drop-down menu. GOLD will not give your information to third parties. All presentations
from the Vancouver family sessions are available for download at www.mpssociety.ca.
Summer 2010
Page 15
Clinical Studies open for enrolment
MORCAP CLINICIAL STUDY FOR
ÉTUDE CLINIQUE DU
MORQUIO SYNDROME TYPE A
SYNDROME DE MORQUIO TYPE A
The Montreal Children’s Hospital is the Canadian site for a
multinational clinical study of Morquio disease type A or
Mucopolysaccharidosis IV type A (MPS IVA) sponsored by
BioMarin Pharmaceutical Inc. The Morquio A Clinical Assessment Program (MorCAP) is designed to provide a fuller
understanding of MPS IVA syndrome in preparation of phase
III clinical trials of a potential treatment.
Patients of all ages are eligible for the MorCAP study. Participation will require a visit of 2 or 3 days to the Montreal
Children’s Hospital in Montreal. Travel and lodging expenses will be reimbursed. No experimental drug is administered during the visit. The study comprises a physical examination of the affected patients, blood and urine tests, a questionnaire concerning medical history, cardiac and respiratory
function tests, and endurance tests.
BioMarin is also conducting a Phase I/II study of an experimental enzyme replacement therapy for Morquio A. The
insights gained from theMorCAP study will help BioMarin
design the phase III trials. More information can be found at
http://www.morquiobmrn.com/ or at www.clinicaltrials.gov
If you are interested in participating in the MorCAP study at
the Montreal Children’s Hospital or if you want more information, contact the study coordinator, Gail Ouellette (email:
[email protected] or phone: 819-543-0550.)
L’Hôpital de Montréal pour enfants est le site canadien pour
une étude clinique internationale de la maladie de Morquio
type A ou mucopolysaccharidose IV type A (MPS IVA). Cette
étude est commanditée par BioMarin Pharmaceutical Inc.
L’étude MorCAP (Morquio A Clinical Assessment Program)
a comme objectif de mieux comprendre le syndrome MPS
IVA en vue d’études cliniques phase III pour un traitement
potentiel.
Les patients de tout âge sont éligibles à l’étude MorCAP.
Leur participation nécessitera une visite de 2 à 3 jours à l’
Hôpital de Montréal pour enfants à Montréal. Tous frais de
transport et d’hébergement seront remboursés. Aucun
médicament expérimental ne sera administré durant cette
visite. L’étude comprend un examen physique, des tests de
sang et d’urine, un questionnaire sur les antécédents
médicaux, des tests de fonction respiratoire et cardiaque et
des tests d’endurance.
BioMarin évalue présentement un médicament expérimental
(thérapie de remplacement enzymatique) dans une étude de
Phase I/II. Les résultats de l’étude MorCAP permettront à
BioMarin de préparer les études cliniques de Phase III pour
ce médicament. Vous pouvez trouver plus d’information sur
ces études à http://www.morquiobmrn.com/ ou
www.clinicaltrials.gov.
Si vous êtes intéressé à participer à l’étude MorCAP à
l’Hôpital de Montréal pour enfants ou si voulez obtenir plus
d’information, contactez Gail Ouellette, coordonnatrice de
l’étude (courriel : [email protected] ou téléphone :
819-543-0550).
Recruitment Open for New Study Led by Dr. Hal Siden
"Charting the Territory: Determining and Documenting Trajectories for Families Where a Child Has a LifeThreatening Condition" is a new multi-centre, longitudinal, descriptive study led by Dr. Rose Steele and Dr. Hal
Siden. Dr. Siden is the Medical Director of Canuck Place Children’s Hospice and a Clinical Associate Professor
at UBC.
This 5-year CIHR-funded study will identify and track children diagnosed with progressive neurological, metabolic, or chromosomal conditions. We will also follow their families to determine and document clinical progression of the child’s condition and the associated experiences of the family. Researchers aim to improve understanding of how to best care for these vulnerable children and their families.
Researchers have begun enrolling families through the Neurology, Biochemical Disease and Genetics clinic at
BC Children’s Hospital. For more information, please visit the study website:
http://www.chartingterritory.com/
Page 16
The Connection
Clinical Studies open for enrolment
questions or sensitive stimuli will be used in the neuropsychological testing. MRI scans do not involve any radiation.
There are no known risks associated with magnetic resonance
scanning itself. We take care not to scan people with some
We are inviting individuals over the age 6 of with MPS I, II or
types of metal in their bodies (from surgery or accident) and
VI to participate in a longitudinal research study seeking to
people with pacemakers or programmable shunts should not
better understand the brain basis for the learning difficulties
enter a magnetic resonance scanning facility. We will ask you
sometimes found in MPS disorders. You or your child must
detailed questions about these risks to ensure that it is safe
be over the age of 6 and able to cooperate in an MRI imaging
for the participant to enter the magnet. The blood draw has
study without any sedation. We are studying the central nervusual risks of bruising but it is only about the amount of two
ous system so that we can better understand the brain
teaspoons.
changes in MPS disorders to find better ways of treating these
problems.
Air and ground transportation and hotel for one night will be
provided to the University of Minnesota, Minneapolis camThe participant will be seen for two sessions in one day, one
pus, for two persons.
for neuropsychological testing that will last about two hours
to three hours, and the other for brain imaging which will last
You may contact Kate Delaney at 612-625-1143
for one hour. Neuropsychological tests will include a brief
([email protected]) or Elsa Shapiro at 612-625-1618
test of cognitive ability and attention, and several tests of
([email protected]) for more information. Thank you for
memory. Some of this testing will be done on a computer.
reading this description
This testing will be done in the Center for Neurobehavioral
Development at the University of Minnesota. You will also
Elsa Shapiro, Ph.D.
come to the Center for Magnetic Resonance Research. The
participant will be placed on a table in the scanner. Imaging Principal Investigator
Professor of Pediatric and Neurology
will require lying still for about 30 to 35 minutes. During
this time the participant can watch a video. We will be doing University of Minnesota
two procedures. One procedure is an MRI that allows us to
calculate the volume of various brain structures (volumetric
******************************
MRI). The other MRI procedure allows us to visualize the
structure in the connections between one part of the brain
Editor’s Note: The Hospital for Sick Children in Toronto will be a
and another (DTI – Diffusion Tensor Imaging). This study
site for this study with Dr. Julian Raiman acting as the Principal
will be different than clinical studies because we are using a
Investigator. Canadian patients are welcome to participate in this
more powerful magnet called a 3 Tesla scanner. Studies with study at the Toronto site or, if more convenient, at the Minneapolis,
a more powerful magnet are more sensitive to the details that Minnesota or Portland, Oregon sites.
we are investigating in this study and are just as safe as less
powerful magnets. We will also draw a small amount of blood
Please contact the Society for more information.
to measure new biomarkers that are being developed by Dr.
Clarke at the University of British Columbia.
Dear adult with or parent of a child with MPS I,
II, or VI:
None of these tests are in any way harmful. No embarrassing
EXPERIENCES OF FAMILIES OF CHILDREN WITH RARE DISORDERS
Principal Investigator: Prof. William H. McKellin, PhD, Anthropology, The University of British Columbia.
Dr. McKellin is inviting families to participate in this study of families of children with rare medical diseases or disorders.
This study aims to hear directly from you about health care, medical research, social services, and community support.
If you live in the Vancouver area and are interested in participating, please contact
Dr. McKellin at 604-822-2756 or [email protected].
Summer 2010
Page 17
MPS Registries & Websites
MPS Registries & Websites:
The MPS I Registry: A resource available for your
physician or health care professional that is dedicated to
improving the understanding of MPS I. With the MPS I
Registry, your physician can access your data and compare it
to aggregate data from around the world. Ask your physician to call 1-800-745-4447, ext.17021 for more information.
The MPS II Registry: The Hunter Outcome Study
(HOS) is a new registry dedicated to patients with MPS II
(Hunter Syndrome). HOS is designed to support the gathering, analysis, reporting and sharing of data from around
the world about Hunter syndrome. More information
about Elaprase and MPS II (Hunter Syndrome) is available
at www.elaprase.com.
The MPS IV Registry: Information about MPS IV
can be found at www.morquio.com. Also available at this
website is the Morquio registry—once registered, it is recommended that updates be made at least yearly, in order to
ensure natural history data is captured to provide evidence
critical for developments of treatments for MPS IV.
MPS Websites:
www.MPSIdisease.com. A Genzyme-developed website
with information and resources on MPS I.
www.Aldurazyme.com. A Genzyme-developed site, providing parents and patients with information on Aldurazyme.
www.lysosomallearning.com. A Genzyme-supported online
resource relating to lysosomal storage disorders (LSDs).
www.MPSII.com. A Shire-developed website, providing
information about MPS II (Hunter Syndrome).
www.MPSVI.com. A BioMarin-developed website, with
information relating to MPS VI.
www.MPSVI.net. Register for free on this site to connect
with your MPS VI community.
www.naglazyme.com. This new Biomarin-sponsored website is now live. You will find expanded content about MPS
VI, its diagnosis and treatment with NAGLAZYME®
(galsulfase) enzyme replacement therapy, and much more.
www.goldinfo.org. Global Organisation for Lysosomal Diseases’ website, offering LSD information and resources, as
well as videotaped presentations from conferences.
Hunter Disease eClinic
This amazing new educational software, developed by Sick
Kids’ Lysosomal Research Group and launched at our International MPS Symposium, is now available through the
Hospital for Sick Children’s website!
The direct links to the educational software are:
Ebook:
http://www.sickkids.ca/research/lysosomalresearchgroup
/contents/Hunter/Hunter_eBook.asp
Eclinic:
http://www.sickkids.ca/research/lysosomalresearchgroup
/contents/Hunter/Hunter.asp
Users need “Flash Player” installed on their computers in
order to be able to view the program, and should be reminded not to use the browser back button.
The Hunter eClinic can also be accessed from the Lysosomal Research Group’s web-site under
"NEW: Educational Resources" on the group home page:
http://www.sickkids.ca/lysosomalresearchgroup/
Check it out!!!!
2010 Summer Studentship Grant Recipients
The Canadian MPS Society is pleased to be funding two Summer Studentship Research Grants in 2010. The Society has a
long history of supporting the research endeavours of talented
university undergraduates and medical students and hopes
these students will go on to successful careers in the MPS or
LSD field. These are the 2010 grant recipients:
Mucopolysaccharidosis Type IIIB (Sanfilippo syndrome):
Conjugation of Naglu to a synthetic protein transduction
domain derived from the HIV-1 transactivator of transcription protein to allow Naglu to cross the BBB

Recipient: Sarah Truelson

Institution: University of Victoria

Supervisor: Dr. Francis Choy

Amount funded: $4,000.00
Chaperone Therapy for MPS IIIC

Recipient: Vincent Boudreau

Institution: University of Montreal

Supervisor: Dr. Alexey Pshezhetsky

Amount funded: $4,000.00
Links to international MPS society websites can be found
on our website: www.mpssociety.ca.
Page 18
The Connection
Research for a Cure: Society-funded Research Projects
Research Report: A comparison of two biomarkers on treatment outcome in
Mucopolysaccharide disease
Principal Investigator: Dr. Brian Bigger
Institution: University of Manchester, Giving for Living Research Centre, Manchester, UK
Funds allocated: $50,000.00 in March 2009
Effective treatment of the Mucopolysaccharidoses (MPS) is
enhanced by early diagnosis and a rapid start to therapy. It is
also important to monitor the success of treatment. Therefore the discovery and evaluation of disease biomarkers for
MPS is essential to clinical management of patients. A test
for MPS I, II and VI used in the clinic involves measuring the
dermatan sulphate:chondroitin sulphate (DS:CS) ratio in
urine. A new technique is detection of heparin cofactor IIthrombin complex (HCII-T), which forms when dermatan
sulphate is present and can be measured using a test called an
ELISA. Using these two biomarkers our aims were to:
currently in submission to Journal of Paediatrics.
We have determined the best ways of collecting and
storing blood samples, which must be taken without an anticoagulant to allow HCII-T detection. Serum samples must
only be tested following one freeze-thaw to avoid complex
deterioration. We have also discovered that HCII-T can be
measured in dried blood spots. Taking blood spot samples is
less invasive than taking serum samples, and measuring HCIIT in blood spots means that this test may be suitable for newborn screening for some types of MPS. However, we have
shown that correct storage of dried blood spot samples is important for HCII-T detection as the HCII-T complex deteriorates in samples that are frozen or left at room temperature.
Reliable measurements can be taken from dried blood spot
samples that have been stored at 4oC for up to one year after
the sample is taken.
We have also shown that HCII-T levels in serum and
in dried blood spots can be used to tell the difference between
MPS I, II and VI patients compared to unaffected individuals,
and to tell the difference between MPS I and unaffected mice.
MPS IIIA and IIIB mice cannot be distinguished from unaffected mice using HCII-T levels, and the difference between
1.
Develop the heparin cofactor II-thrombin (HCII-T) HCII-T in MPS III and IV patients and unaffected individuals
biomarker for analysis of dried blood spots in patients and is very small. Therefore we suggest that HCII-T will be a usemouse models.
ful biomarker for diagnosis of MPS I, II and VI, but not MPS
III or IV. When patients receive bone marrow transplanta2.
Compare the HCII-T biomarker with the existing
tion and/or enzyme replacement therapy we have observed
DS:CS ratio biomarker for efficacy at diagnosing treatment
that both the DS:CS ratio and HCII-T levels decrease in reoutcome following therapy in MPS patients.
sponse to treatment. However, when treatment is changed or
We have achieved both of these aims, and have published
a patient is not responding well we see a marked increase in
results regarding evaluation of the HCII-T biomarker in the
HCII-T, but not in DS:CS ratio. Therefore the decrease in
mouse models of MPS I, IIIA and IIIB and the analysis of
disease biomarkers matches the clinical improvement seen in
HCII-T in blood spots. The results from our comparison of
these patients, and HCII-T appears to be particularly responHCII-T and DS:CS ratio in patients undergoing treatment are sive to acute changes in treatment response.
The Canadian MPS Society's 2010
Research Grant
2010 CIHR SHOPP Program Partnership
Grant:
The Canadian MPS Society is pleased to announce it is partnering with Canadian Institute of Health Research (CIHR) to
fund a fellowship into Lysosomal Disease Research. The felComprehensive study of pathogenic glycosaminoglycan stor- lowship is for 3 years and the Society and CIHR are both
funding half of the $135,000.00 award (45,000.00 per year
age in a murine model of MPS I
for 3 years):

Principal Investigator: Dr. Lorne Clarke
The Society is pleased to announce the recipient of its 2010
Research grant:

Institution: University of British Columbia, Vancouver, BC
Funds allocated: $100,000.00 (two year grant:
$50,000.00 per year for two years)
The molecular mechanisms of microgliosis in GM2 ganglioside lysosomal storage diseases


Recipient: Elizabeth J. White

Institution: McMaster University

Supervisor: Suleiman A. Igdoura
Please visit our website and click on “research” to read the full

Funds allocated: $67,500.00 (22,500.00 for 3 years)
research project summary.
Summer 2010
Page 19
Fundraising—The 2010 MPS CUP
On May 15th, 2010 the puck
dropped for the 8th annual
MPS CUP Fantasy Hockey
Game. Players and fans celebrated International MPS
Awareness Day at the North
Shore Winter Club in North
Vancouver, where former Olympian Cammi Granato showed
the guys a thing or two and everyone displayed Olympic spirit
by “believing” in a common
goal—beating MPS.
Following the game,
players and others in their evening finery arrived at the Sutton
Place Hotel Vancouver to listen
to Yvonne McSkimming’s incredible cocktail hour performance and bid on the wide array
of auction items. After a gourmet dinner, the highlight of the Avery Purcell, brother of Trey (MPS II), drops the puck at the 2010 MPS CUP
evening (besides the chocoholic
as North Vancouver Mayor Richard Walton & MP Andrew Saxton face off
dessert buffet!) was a talk by
Mark Dant of the Ryan Foundation. Mark touched
the crowd with his family’s story of courage and perseverance and their—and their donors’—enormous impact on the lives of those with MPS. Mark made us
feel as if we could collectively do so much more than
we already have for those suffering with MPS and
related diseases, and by the end of the night we had
raised close to $96,000.00.
It’s always almost impossible to express our
gratitude toward all the people who make this event
happen, but from the bottom of our hearts, thank you
so much to our entire MPS Cup team.
Kirsten and Todd Harkins
Kirsten, Todd, and honourary MPS CUP
teammate Mark Dant at the 2010 game
Page 20
The Connection
Clockwise, from top left:
Gary Lewis & Greg Wolfe
flank hockey great Paul
Reinhart; Brian Dunster
& ex-Canuck Chris Oddliefson wear the stripes;
goalies Shazan Hemani &
Nicklas Harkins strategize before the game;
Cliff Ronning gives Garry
Valk some advice; Global
TV’s Steve Darling with
the NSWC’s NHL alumnus Randy Heath & Darwin Schandor; Grant
Stewart, Stanley Cup
winner Bill Ranford, Glen
Barker and Lee Coonfer
relax and tell some
hockey stories between
periods in the white
team’s dressing room.
Kirsten Harkins & game-day coordinator Kelly Tait with the trophy
Summer 2010
Fans (including the Harkins & Purcell
families) cheer on team MPS
Page 21
MPS CUP Donors
$5000
Vidalin Family Foundation
Ledcor Construction Limited
$1000—$2000
Southwest Contracting Ltd.
Kirsten & Todd Harkins
National Concrete
Columbus Meats
MDE Mechanical
ECM Espresso Machine
$500—$1000
Imperial Paving
Wade Bartok
Bernie & Christie Geiss
John & Laura Swift
Dan & Sandi Turner
Perimeter Mini Bins Ltd
More donations..thank you!
Leslie & James Carter
D & L Douglas
PPI Financial Group
Jordan Weal
Wade Bartok
Kenneth Dixon
Gregory & Kelly Wolfe
Kyle Smith
Michael Urbani & Lisa Dumbrell
Horizon Engineering Inc.
K & A McBurney
John & Joanne Marquis
Darcy Knox Agency Ltd.
Ryan & Deb Purcell
Neil Stevenson-Moore
Steve and Sandy Frasher
Page 22
John & Joanne Marquis
Bob Foulkes
Gary & Jill Ley
Jacob Heilbron & Heidi Castle
Ken Huckell & Joanne Maltby
Mike Allen
Mike & Mary Ann Nightingale
Andrew Saxton MP
Lloyd Blackburn
Julia & John Gerbrecht
Josee & Graham Wilson
Rick & Anna Marks
Scott & Sue-Ann MacCara
Joe Baudash & Marnie Gittens
Karen & Tim Saxvik
Hugh Lewis
Jonathan Calvano
Brent Malcolm & Jennifer Clifton
Ron & Christy Andersen
Cara Wilson-Haffenden
Dan & Tina Priest
Kent Scarborough
Derek & Rhonda Steinke
Lynn& Mark Pickering
Guy Holeksa & Karen Arnold
Darren Samms
Pam & Chuck Vidalin
Rob Thomson & Marianne StoweThomson
Gary & Liz Mallender
Dr. Mark Olesen Inc.
Tim & Sheila Keon
Megan Ryan
Janet & John Robinson
John & Laura Swift
Jenifer Gentle & Hans Hamberger
Caulinda & Wade Bartok
Ken & Helga Irving
Brenda MacLean
Gregory & Kelly Wolfe
Fiona Douglas-Crampton
Carole & Kent Elliott
Mary Elliott
Langton & Carol Lynn
Jennifer Griffiths
Cindy Yam
Kim Lina
Matreya Monro
Marie Muniz
Kelly & Shelley Weal
Lisa Tomlinson
Lisa Ross
Dave & Lola Tomlinson
Julie Coghlan-Smith
The Connection
Thanks for pledging our players!
$1500
Peter & Ellen Muirhead
$500
Earl & Barbara Phillips
Nancy Trott & Ian Hanoomansingh
Linda & Graham Brown
Roger Taplin
Robert Toombs
Elizabeth Vogt
Gysbertus Warmerdam
Dawn Ann Webster
Cindy Yam
Vincent Yip
James & Jen-Beth Fulton
Rita Gacina
Paul Grech
Trish Ousted
Colleen Griffin
Richard Balfour
Kevin Gulstene
Steven Balogh
Kirsten & Todd Harkins
Amy Barr
I Hsin Tng Hsu
Judy & Terry Byrne
Linda Jensen
Yin-Wei Chiu
Hwan Han & Suk Cho Jung
Stephanie Dong & Ian Gillespie
Laura Kaplan
Elizabeth Fawcett
Jakob Kwist
Jeroen Kwist
Dong Hyun Lee
Katherine Lin
Lina Kim
Jean Liu
Ana Lopez
Patricia MacDonald
Hope & Dave Madsen
Lisa Martz
Yvonne Nimmo
Donna Pistilli
Opposite page, top to bottom: Fiona DouglasDonovan Plomp
Crampton & Carrie Nimmo welcome guests to the
Shelley Potratz
Gala; Nicklas Harkins & friend Cole Todd check
out the silent auction items; Dr. Sylvia Stockler & James Roth
husband Dr. Osman Ipsiroglu with Kirsten;
Kirsten with outstanding MC, CTV’s Mike Killeen
& his wife Jill; North Vancouver MP Andrew
Saxton with fabulous Canadian MPS Society employee Jill Ley & her husband Gary.
This page, top to bottom: BJ MacDonald, Kirsten,
Lisa MacDonald, Gillian Olesen & Christy Andersen enjoy Yvonne McSkimming’s cocktail reception performance; Kirk Mclean flips a coin during
the 50/50; Krista Nickel wins the pot but generously donates it back; Gary Lewis & Lee Coonfer
from Ledcor flank Ivan Harmatny from Lower
Mainland Steel—both companies announced
substantial donations; Keynote speaker Mark
Dant (3rd from left) with grateful parents of kids
with MPS: Hans Hamberger & Jenifer Gentle,
Kirsten & Todd Harkins, and Tina & Dan Priest.
More pledges
Summer 2010
Page 23
Thank You Auction Donors!
A Woman's Touch Housecleaning
Bill Adams
James Adamson
Air Canada
Luciana Alvarez
Arts Club Theatre Company
Deanna Barlow
Batsch Group Inc
Black Gold Lodge
Bosa Foods
Chris & Cindy Bouchard
Barb Brebner
Shelly Bryant
Canucks Alumni Association
Canvas Salon
Leslie Carter
Centennial Theatre
Carmen Chou
Cove Watersports Inc.
James Cronk & Theatre Under the Stars
Curves North Vancouver
Darcy & Madeline's Agency
Deep Cove Canoe And Kayak Centre
EA Canada
Escape Hair & Body
Escents Aromatherapy
Fine Science Tools Inc
First Impression Hair Design
Floe Fitness
Bob Foulkes
Genzyme Corporation
Julia & John Gerbrecht
Grouse Mountain Resorts
Neal Harkins
Todd Harkins
Vera Hay
Carolyn Hubbard
Mike & Lisa Hudson
JC Studio
JCA Communications Inc.
Jesse Smith - Designed In-House
John Henry Bikes
Linda Jones
Kevin Kerr
Kim Kirk
La La Home Decor
La Notte Ristorante Italiano
Teresa Lake & Christopher Burt
Land Rover
Page 24
Christina Langille
Level 10 Fitness (NSWC)
Gary Ley
Lynn Valley Optometry
Maa Yoga
Hockey-sockey creator Megan
Lloyd with her husband Neil
Red Tulip Gift Gallery
Rower's Cove
Sally Sheppard Garden Designs
Sandy Elliott Agencies Ltd.
Andrew Saxton MP
Kent Scarborough
Seymour Golf & Country Club
Marie Sherk
Shimano Canada
So.Cial Restaurant & Oyster Bar
Spa Utopia & Salons
Sutton Place Hotel Vancouver
TBG The Bicycle Group Inc. (Kona
Bicycle Co.)
The Destination Slope & Surf Outfitters
The Mystic Wardrobe
The Vancouver Club
Thomas Haas
Time Out Sports
Tourism Vancouver
Vancouver Aquarium
Vancouver Board Of Trade
Tracy Majhen
Maplewood Farm
Terri & Dave Margo
Marine Harvest
Mayfair Lakes Golf Course
Mercedes-Benz Canada Inc.
Museum of Vancouver
Naa Sheka Clothing
(West African Fashion)
Dan Urbani & New
Petcetera Retail Limited
NHL Alumni
NHLPA
Nintendo of Canada
Ltd.
North Shore Athletics
Committee members Karen Saxvik, Katrina StobNorth Shore Jazz
bart, MaryAnn Nightingale & Julia Gerbrecht
North Shore Pest
Detective Ltd.
Vancouver Canadians
North Shore Pilates Now
Vancouver Canucks
North Shore Sports Medicine Clinic
Vancouver Chamber Choir
North Shore Triathlon Club
Vancouver International Film Festival
North Shore Winter Club
Vancouver International Fringe Festival
North Vancouver Rec Commission
Vancouver International Writers
Northlands Golf Course
Festival
Oceaner Sporting Goods Canada Ltd.
Vancouver Recital Society
Mark & Gill Olesen
Richard & Susan Vann
Charles Osieja & Jenny Cleary
Mendel Vysholid
PCI Developments Corp
Wendy Walker
Playhouse Theatre Company
Josee & Graham Wilson
Pro-formance Goalie Schools
Y Yoga
Ravens in Motion
Easton—Chris Zimmerman
RBC Financial
Parkgate Safeway—”Believe” cookies
Real Hip
The Connection
The MPS CUP would not be possible without the huge contributions of our entire team.
Thank you so much to the players, sponsors, committee members, volunteers and donors of all kinds
for believing in our vision of a brighter future for Canadians with MPS.
Thank you to our
2010 MPS CUP
celebrity players:
Dave Babych
Terry Bingley
Steve Darling
Stefan Elliott
Cammi Granato
Todd Harkins
Randy Heath
Shazan Hemani
BJ MacDonald
Kirk McLean
Bill Ranford
Paul Reinhart
Cliff Ronning
Dave Tomlinson
Garry Valk
Wayne Van Dorp
Jordan Weal
Andrew Saxton, MP—North Vancouver
Richard Walton, Mayor—North
Vancouver
Thank you to our amazing volunteers:
Shelly Bryant
Carrie Marshall
James Carter
Sam Chernomaz
Cara Wilson-Haffenden
Summer 2010
Raquel Dominguez
Julia Dapavo
Brian Dunster
Chris Oddleifson
Perry Martin
Chris Todd
Steve Tait
Gary Krogfoss
Rick Marks
Cameron Marks
Nicklas and Jonas Harkins
Maya Andersen
Shaelyn Tait
St. John Ambulance volunteers
Ashley Lloyd
Megan Lloyd
Carrie Nimmo
Karen Issigonis
Ali Maclean
Kay Fulford
Paul Fulford
Chloe Ley
Lauren Overholt
Fiona Douglas-Crampton
Tami Perkins
Brad Shapendonk
Clinton Mulligan
Yvonne McSkimming & friend
Tony Scott, our auctioneer!
Heart-felt gratitude to our very dedicated & enthusiastic committee:
Christy Andersen
Leslie Carter
Heidi Castle
Julia Gerbrecht
Lyle Grant
Kirsten Harkins
Todd Harkins
Christina Langille
Joanne Maltby
MaryAnn Nightingale
Gillian Olesen
Karen Saxvik
Katrina Stobbart
Kelly Tait
Garry Valk
Gala volunteers Ali Maclean, Christina
Langille, Kay Fulford & Sam Chernomaz
Page 25
Fundraising—Tacos for Trey
Tacos for Trey was held May 8th,
2010 and brought together kids of all
ages for an afternoon of balloon twisting, face painting, a raffle and silent
auction, and of course a piñata and
lots of tacos! Deb Purcell tells us
what the event meant to her:
At ‘Tacos for Trey’ yesterday, people asked me how I was doing, how I thought the event was
going, and I was unable to comment in any real kind of way.
Being at ‘my’ event was an out of body experience. This event
that I have been planning since September, that I have neglected and yelled at my kids because of, that I’m planning
because I want to save my child’s life, that I have poured my
heart and soul and everything I am and hope to be into…
when it actually came, it was surreal.
pouring rain, all the kids on the street come out to play. We
LOVE our street. Even dads whose kids are grown and have
moved on, come out to play. Well, our neighbors came. We
could have had a block party at T4T. They ALL came. And
Trey’s T-ball team and soccer team came too. And that brings
me to tears just thinking about it. How much better can your
community get than when your entire block of friends and
sports teams show up to support Trey and our family.
Because, to be honest, lots of people don’t understand or are
uncomfortable or nervous about or scared of difference. And
that makes me hesitate when I register Trey for activities or
when we go out. Will they be receptive? Will they make negative comments behind our backs or to our faces? Will they
‘get’ us enough or be open enough to become our friends and
look past our differences?
So, all my questions and wondering about how and if we will
be accepted, were answered yesterday. And it was a yes. A
Looking out over the hundreds of people who came to the
resounding yes. This is where I want to live- a place that is
event was unbelievable. We’ve come to have regulars. Our
family and close friends who have supported us the whole way willing to get to know your family, and go to bat for them,
through. People who we may not know quite as well, but for regardless of all its differences. Because we need that. And it’s
some reason, know we need their support and have really big not easy to find. So thank you neighbors and community, for
hearts or have been through something themselves so under- embracing us. You have no idea how much it means (if you
stand what we’re going through. They’ve helped us grow and were sitting here watching tears roll down my face while I
type, you might have a better idea).
watch us grow our event from our home in its first year, to
the school for the past 2 years. A
And now I have about 100 other
lot of these ‘regulars’ I see once
stories I’d like to tell about those
or twice a year or haven’t even
who came and those who
seen them since the last T4T,
touched me and those who lifted
and that brings me to tears beme up. But then I would be writcause even though I don’t see
ing a thesis.
them or talk to them or thank
I said yesterday, when I went up
them or hug them, they still
to say my thank yous at the mic,
show up.
that although we may not be able
Grizz and Aimee also blew me
to thank each of you individually,
away. Since beginning our MPS
we do want you to know how
II Research Fund in 2007, T4T
grateful we are. We hope you
(and ‘Time for a Cure’ last year)
know. Because we do not want
has been the only fundraiser to raise money for our fund.
ANYONE who has supported us, in the hundreds of ways
Then yesterday, Grizz, one of Ryan’s friends from work went there are to support us, to think we are ungrateful. You are
up to the mic and presented us a cheque for over $2,000
literally helping us save our son’s life. I cannot think of anyraised at a poker night last weekend.
thing that is more meaningful to us than that. So please know
that none of your support will be forgotten or taken for
And then Aimee came to talk to me. She and her husband
put on a yearly charity fundraiser. In the past, proceeds have granted.
always gone to the same non profit. For their upcoming
event, however, they have decided to split the charitable proceeds, with 1/2 going to our research fund.
Got hope? You bet. Thank you for helping us hold onto it.
And to make it even better, Tacos for Trey, was a HUGE success. We raised $24,800 on the day, $38,900 in total, with a
And then this year, our community came. In the past, we
few donations still coming in. To give you an idea, the first
haven’t really had this. And when we moved to Lynn Valley
year we raised $17,000 on the day, $30,000 in total. The seclast June, we came here because we were looking for commu- ond year we raised $18,000 on the day, $32,000 in total. We
nity. That was incredibly heartwarming, to put it in a not so
raised wayyyyyyy more than the past. And this means we can
teary way. We live on a cul-de-sac, where, if it’s not freezing or fund a research grant!! Cure, here we come!
Page 26
The Connection
Thank you to the following MPS II
Research Fund donors:
Spiro Karfakis
Ryan Anderson
Shirley Anderson
Tracey Arnish
Karen Berkhout
David Blue
Andrea Brann
Steve Adelson
Melissa Burrowes
Pauline Campbell
Clara Campedelli
Francis & Bonnie Caouette
Simon Carlsen
Judy Carter
Raimey Casiro
Sue Cech
Ilse Cehak
Kurt & Erica Cehak
Michelle Cehak & Tom Daycock
Ian & Peter Cicansky
Corintia Developments Limited
Leane Dalgleish
Robert & Leigh Anne D'Angelo
Maureen Dockendorf
Mary Elliott
Sanaz Farkhad
Sheila Fee
Marguerite Ford
Deborah Frisk
G.F.H. Enterprises Ltd.
David & Susan Geddes
Sandra Goodall
Maryanne Gosling
Teresa Grandinetti
Doug Green
Deborah Gregg
Jesse Gregson
David Griffiths
Karen Harvey
Dr. and Mrs. I.N. & Patricia Henderson
William & Pat Hesketh
Nancy Hill
Robert Holbrook
P.A. Holt
Gordon & Sandra Hood
Russell Horwitz
Julia & Douglas Huie
Summer 2010
Margaret Huntingford
Chantal Iorio
Joan Jamieson
Jill MacLauchlan
Leonor Johnson
Laurie Lee Jones
Simon Jori
Britta Joslyn
Peter & Charis Kalesnikoff
Larry & Shirley Kancs
Jacki Kirkwood
Ilse Kluge
Wolfgang Kluge
Joslin Kobylka
Sheila Koeller
Darrell & Kathie Krahn
Guy Laboissonniere
Carmen Lansdowne
Robert Ledingham
Joni Low
A.L. Maberley M.D.
Gina Macdonald
James & Susanne MacDonald
David Magee
Howard & Caroline Malm
Judy May
Laura Mazur
Mrs. Christine Mills
Nicole Moon
Jordann Nettles
Lara Ng
North Shore Credit Union
Haley Oxenham
Jorge & Vera Paclawsky
Rudy Pagliaro
Harry & Mimi Philipp
Dan & Tina Priest
Zdenka Prunkl
Gayle Purcell
Ryan & Deb Purcell
Michelle Reining
Amber & Richard Rennie
James & Milena Robertson
Raelyne Romman
Jamie Ross
Dene Rossouw
Caroline Rowles
James Salberg
Jack & Edna Sandberg
Terra Scheer
Harald & Christina Schnetzler
John & Marlene Schreiner
Glendine & Ronald Schultz
Scotiabank
Carole Segal
Sico Cinematic Inc.
Dorothy Silvestre
Pavel Siska
Ross & L. Diane Smith
Jennifer Spence
Dr. Victor Spielmann
Pat Steeves
The Talking Picture Company Inc.
Sean Tobin
Lynne & Jim Tomlinson
Lori Turk
Manon Vrain
Brian Wales
Dorothy Watters
Kyle Wheatley
Shauna Whittall
Peter & Sharon Willemse
Darren Yada
Linda Yada
Ryan Yada
Dr. Jack Yasayko
Kelly & Chris Zimmer
Emma Zolbrod
MCM Motion Photography Ltd.
More to come in
our fall edition
of the Connection!
Page 27
THANK YOU TO OUR FABULOUS TACOS FOR TREY VOLUNTEERS
Jennifer Clifton
Lina Wallman
Lisa Odland
Michele Griffin
Kay Fulford
Courtney Inman
Val Ramsey
Aunty Nancy
Aunty Lynda
Sheila Weckerle
Meaghan Grant
Maria Lutz
Janice Springford
Ayasha Valji
Rebecca Chapple
Nabil Valji
Fiona Petigra
Anne-Sophie Rodet
Tracy Souza
Chloe Ley
Daianne Fernandes
Hiroko Araki
Page 28
Michiko Araki
David Sandberg
Dino Goundouvas
Sarah Little
Charis Kalesnikoff
Kirsten Harkins
Nicklas Harkins
Jansen Harkins
Sandy Kirkwood
Neil Whittaker
Dorothy Watters
Leora
Alysha Radvak
Shayna
Robyn Kanks
Brook Ewert
Ed Peterson
Erika
Katy Colby
Mona Sabzevari
The Connection
Fundraising–Sixteen Candles for Elisa
Candles and hearts were aglow for Elisa Linton’s Sweet Sixteenth at the Sanfilippo Children’s
Research Foundation’s Annual “Evening for Elisa” Gala Dinner and Auction
For most teenagers, a 16th birthday marks a
milestone. For a child affected with Sanfilippo, 16 marks a miracle.
Elisa Linton, the inspiration behind The Sanfilippo Children’s Research
Foundation (www.alifeforelisa.org), turned
16 this March, and the SCRF celebrated by
theming their annual gala “16 Candles for
Elisa” —a momentous event for the Linton
family and their supporters.
The event took place on Thursday, March 25th, 2010 at
the Paramount Banquet Hall for the 700 guests and volunteers
and it was graciously emcee’d by the stunning Anne Marie Mediwake of CBC News Network. Many MPS families were in attendance.
There were candles of celebration for Elisa who was pretty in pink and was able to attend her Sweet 16th. The centrepieces were birthday cakes, festive with flowers and candles. As
guests lit the candles and sang Happy Birthday, everyone made a wish for Elisa and Sanfilippo
children everywhere.
There were also candles of hope alight that night—hope that Elisa will continue to beat
the odds with her indomitable spirit, and that the SCRF will find a cure for her and many other
children like her. Altogether, the event raised $195,000—enough to fund two research projects
for an entire year, keeping dedicated researchers working on a cure and treatment for Sanfilippo
children.
The Linton family extends their thanks to all the generous companies, guests and volunteers who shared their time, talents and financial resources in many ways to help make this
evening a spectacular success. In turn, the Canadian MPS Society thanks the Linton family for
the courage, faith, sacrifice and energy they have demonstrated in uniting so many people in their life-saving mission and for
raising hundreds of thousands of dollars for MPS III research. Watch for news of the SCRF’s next gala in the spring of 2011.
Above: Elisa celebrating her 16th Birthday with her family and loved ones
Fundraising—Just Singin’ Round
The Canadian MPS Society was the featured charity at the April 6th, 2010 Just Singin’ Round singer-songwriter showcase at
the Vancouver Rowing Club in Stanley Park. This is the fifth year we will receive a portion of the funds raised from the year’s
showcases, and we thank the Synergy Foundation for their support, the musicians for donating their time and talent, and
everyone who came out to listen
to the fabulous music! Join us
for more JSR events. Check our
website for upcoming dates.
Far left: Mark James
Fortin, Yvonne McSkimming, Beverley Elliott &
Pierre Charlebois perform;
left: Adam Woodall, Don
Alder & Karen Laursen
Summer 2010
Page 29
Fundraising– Canadian MPS Jeans Day
Help bring awareness of
MPS Diseases into your
community!
Hold a Canadian MPS Jeans Day
on October 8th—or on any day of
the year— just follow these
simple steps:
 Submit a proposal to your school's principal or your employer **email Kirsten
([email protected]) or Jill ([email protected]) for a word template**
 Email or call the head office to let us know how many people you anticipate will be participating.
You will receive a Canadian MPS Jeans Day package with enough stickers for your event. Packages
will also contain 2 posters, 5 colour brochures (you will have to photocopy enough brochures to distribute at your event), and a donor information sheet to record the names and addresses of those
wishing to receive income-tax receipts for donations of $10.00 and over.
 Fill in the event date and expected donation amount on your posters (the expected donation is up
to your discretion depending on the location: we recommend between $1.00—$5.00) and advertise
Canadian MPS Jeans Day at your school or workplace. Download an 8x11 poster PDF from our
website if you would like to print out additional posters.
 Organize volunteers to collect donations and distribute stickers and brochures on the morning of
your event.
 Ensure those who donate $10.00 or more fill out the donor form completely if they wish to receive
income tax receipts.
 Take a photo at your event to share with our members in the next issue of the Connection!
 Submit your donations, donor forms, photos, and a brief write-up, with photos if possible, about
your event within 30 days of the event date.
Canadian MPS Jeans Days are easy to hold and are excellent opportunities to raise awareness and funds
for the society. Many of our members have found them to be extraordinarily helpful in educating
school communities about our children's unique conditions. Please consider holding a Canadian MPS
Jeans Day at your child's school or at your workplace.
Page 30
The Connection
On-going Fundraising
On-going Fundraisers:
More Great MPS Fundraising Ideas:
Used Stamps:

Sign up through your United Way Donor Choice
Program. Designate The Canadian MPS Society as your
“OTHER” choice by providing our name, address and
Charity # 129030409 RR0001. Double your support by
enrolling the Society in matching gift programs, available through many companies.

Organize a walk or run

Have a garage sale

Organize a Sports Tournament

Participate in Craft Sales

Organize a Canadian MPS Jeans Day

Host a party: i.e. Pampered Chef, Discovery Toys,
Partylite.

Organize a dinner or barbeque

Remember, when you sponsor a fundraiser you are not
only raising money to help find a cure but you’re also
educating the public about MPS.
Please save your stamps and mail to our stamp collector:
Laura Wassilyn
5 Kitson Drive
Scarborough, ON M1M 3C7
TIPS ON MAILING STAMPS:
Please trim stamps leaving 1/4
inch of paper around them. Any with tape or damaged perforations should NOT be submitted as they cannot be sold.
Thanks for your support!
Fundraising Reminders:
Contact the office and request an event proposal form.
Fill out and submit the form so that the head office knows
the details regarding the events you are holding.
Don’t forget to submit a brief article, with photos if possible,
for the Connection about your fundraising success stories and
suggestions—they are terrific resources for other families planning events.
Thanks for your enthusiastic support!
FUNDRAISER COOKBOOK
Board Member Mary Nelis, mother of Monika Nelis-Dupont (MPS I HS), is putting together a
recipe book to raise funds for the Canadian MPS Society, but she needs your help! Please contribute by sending your favourite recipes, especially your favourite barbequing & grilling recipes, to
Mary at [email protected], or, if you prefer, you can send or email them to the Canadian
MPS Society & we will forward them. Mary hopes to have the cookbook ready for sale this
summer.
Do you need respite care?
Caring for an exceptional child can be exhausting—The Maxwell
Alexander Settari Fund has generously donated funds to our
Family Assistance Program specifically for respite care.
Interested members should submit a Family Assistance Program
application form, found on page 34, and contact Kirsten for guidelines.
Summer 2010
Page 31
Society Merchandise
CANADIAN MPS SOCIETY GOLF SHIRTS!
AVAILABLE IN MEN’S XXL, XL, L, M, & S, AND WOMEN’S
(fitted style with buttons) L, M, & S.
$40.00 (includes shipping)
ALSO AVAILABLE ARE A LIMITED NUMBER OF CHILDREN’S T-SHIRTS
(These shirts come in Youth L, M and S and are for sale for $12.50 (including shipping).
TO ORDER, visit our online store at www.mpssociety.ca
or call us at 604-924-5130 or on our toll free number 1-800-667-1846
NEW! “Expression of Hope II”
art cards are now available!
These inspiring cards come in a pack
of 20 original designs (including the
beautiful piece to the left).
View all selected pieces as well as all
submissions to Expression of Hope II at
www.expressionofhope.com.
Artist: Zack
Country: USA
Condition: Pompe
Order your art cards for $25.00 per package
(including shipping) by visiting our online
store at www.mpssociety.ca, or calling
604-924-5130 or 1-800-667-1846.
I picked this flower to paint because I
thought it was kinda like me. It's calm,
cheerful and it's not afraid to take up a lot
of space. I liked it a lot.
ORDER YOUR
CANADIAN MPS SOCIETY
AWARENESS BRACELETS!
Our bracelets are royal blue and inscribed with “BELIEVE - MPSSOCIETY.CA”
Our awareness bracelets come in three sizes: large, medium, and small and are available
for a minimum donation of $2.00 per bracelet, plus shipping.
Please place your order through our online store at www.mpssociety.ca
or call us at 604-924-5130 or 1-800-667-1846 and help spread the word about MPS diseases!
Page 32
The Connection
The Canadian Society for Mucopolysaccharide & Related Diseases Inc.
2010 Membership Form
To register or renew your membership, please submit this form or go towww.mpssociety.ca (please log in to your account when renewing)
First/Last Names: _________________________________________________________________________________
Address: _______________________________________________________________________________________
City: __________________________________ Province: _________________ Postal Code: __________________
Telephone: ________________________________ E-mail: ______________________________________________
Affected Family:
Relative/Professional/Other:
Out of Country:
______$25.00
______$30.00
______$35.00
AFFECTED CHILD’S NAME(S) AND
Enclosed membership payment:
Enclosed donation:
Total:
DATE OF BIRTH
MEMORIAL DATE
$ ________
$ ________
$ ________
SEX
SIBLINGS’ NAMES
DIAGNOSIS/
MPS DISORDER
Would you like your name to appear in our Parent Referral Directory? Yes ______
No ______ Initial consent:__________
Would you like to have your affected child’s name appear on the Birthday/Memorial page? Yes ______ No ______
If so, please fill in all information in the above chart, as you would like it to appear.
I am willing to allow the Society to publish pictures of my living/deceased child(ren) for posters, advertising, booklets, brochures,
and the MPS website. Yes ______ No ______ (Please send/email clear photos. We will return photos if requested.)
Signed:______________________________________________ Date:_____________________
Please send information on MPS Diseases to the following professionals:
Name:_______________________________________________________________
Address:_____________________________________________________________
IF YOU HAVE AN AFFECTED CHILD AND CANNOT AFFORD THE $25.00 MEMBERSHIP FEE,
PLEASE INFORM THE OFFICE AND WE WILL SEND YOU COMPLIMENTARY NEWSLETTERS.
The Canadian MPS Society is committed to providing support to fami- Please make cheques payable to:
lies affected with MPS and related diseases, educating medical profesTHE CANADIAN MPS SOCIETY
sionals and the general public about MPS, and funding research so
that one day cures will be found for all types of MPS and related disPO Box 30034, RPO Parkgate
eases. Members will receive:







Four newsletters per year (Spring, Summer, Fall, Winter)
Our Family Referral Directory
Our Annual Report
Access to our Family Assistance Program
Advocacy support
All new publications printed by the Society
Invitations to attend family meetings and conferences
Summer 2010
Please charge my credit card in the amount of $______________
Credit Card #________________________________________
Name on card ________________________________________
Expiry Date __________Signature________________________
Registered Charity # 12903 0409 RR0001
Page 33
FAMILY ASSISTANCE PROGRAM
FUNDING APPLICATION
Date:_________________
Name:________________________________________________________________________
Address:_____________________________________________________________________
_____________________________________________________________________________
Phone: ___________________________________
Email: ___________________________________
Name(s) of affected child(ren):___________________________________________________
Amount of funding requested: ______________
Please describe what the funds will be used for: ____________________________________
_____________________________________________________________________________
_____________________________________________________________________________
_____________________________________________________________________________
Will the funds being requested be used to pay for part of a larger project or piece of equipment?
(eg. home renovations, wheelchair-accessible van, etc.)
Yes________No_________
If you answered yes to the previous question:
What is the estimated total cost of the project/equipment? _____________
Please list other funding agencies you have applied to for funding for this project/equipment,
and funds received, if any:
_______________________________________________________________________
_______________________________________________________________________
□ Estimate(s) included. (Please see guidelines for information on required estimates.)
Signature______________________________________________Date___________________
Thank you for submitting your Family Assistance Program funding application. The Executive Director or a member of the Board of
Directors will contact you as soon as possible to inform you of the Board’s decision regarding your application.
Please visit www.mpssociety.ca or email [email protected] for complete Family Assistance Program guidelines.
Page 34
The Connection
The Canadian MPS Society’s Publications
Canadian MPS Society booklets and merchandise can now also be ordered through our online store at www.mpssociety.ca
NUMBER
OF COPIES
NAME OF BOOKLET
PRICE
PER COPY
Family Resource Book - English or French*
4.00
Daily Living with MPS and Related Diseases
2.00
MPS I (Hurler/Scheie Diseases) - English or French *
2.00
MPS II (Hunter Syndrome)
2.00
MPS III (Sanfilippo Syndrome)
2.00
MPS IV (Morquio Syndrome)
2.00
MPS VI (Maroteaux-Lamy Syndrome)
1.00
Mucolipidosis II (I-Cell Disease) and III
1.00
Fucosidosis
1.00
Mannosidosis
1.00
Aspartylglucosaminuria
1.00
Mucolipidosis IV
1.00
Multiple Sulfphatase Deficiency
1.00
Tay-sachs & Sandhoff Disease
1.00
Hearing impairment in MPS Children
1.00
Is your child Having an Anaesthetic?
1.00
Bone Marrow Transplants in MPS Children
1.00
The Pattern of Inheritance
4.00
Choices – When your child is Dying - English or French*
7.00
Video—A Roll of the Dice
10.00
TOTAL
PRICE
*All booklets are in English with the exception of the Family Resource Book, MPS I Booklet & Choices. Please circle language.
PLEASE ADD $3.00 FOR SHIPPING. (For 6 or more booklets add $10.00.)
Name: __________________________________________________________________________________________________
Address: ________________________________________________________________________________________________
City:______________________________________________Province ________________Postal Code: ____________________
Telephone: ( _____) _________________
# of Booklets:
_________________
Shipping & Handling: _________________
Send your order and cheque to:
The Canadian MPS Society
For payment by credit card, please call our head
office at (604) 924-5130 or 1-800-667-1846.
Summer 2010
Page 35
S
MPS Society Logo Wear

Indigo Sweatshirts
$30.00

Jean Shirts
$30.00

Indigo T-shirts
$20.00

Children’s t-shirts
$15.00
M
L
XL
Plus $5.00 for shipping & handling (per item)
Total Enclosed__________
Page 35
JOIN TEAM MPS FOR THE SCOTIABANK
TORONTO WATERFRONT MARATHON!
Sunday, September 26, 2010
5 km, 1/2 marathon, full marathon
Contact Judy at [email protected] and join our team!
The Canadian MPS Society is an official charity with the Scotiabank Toronto Waterfront Marathon.
Collect pledge donations of at least $50 and we will provide your registration and a team t-shirt.
Tel: (604) 924-5130 / 1-800-667-1846
Fax: (604) 924-5131
www.mpssociety.ca

Summer 2010 - The Canadian MPS Society

Transcription

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