Treating the Whole Disease

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Treating the Whole Disease
Duchenne muscular dystrophy
T reating the W hole Disease
DUCHENNE IMPACTS:
1 in every
3,500 live male births
What Is It?
is the most common and lethal
muscle disease in boys.
24,000
300,000
boys in the U.S.
boys worldwide
Impact on the Body
DUCHENNE IS A PROGRESSIVE,
MUSCLE-WASTING DISE ASE.
It results from a defective gene responsible for
producing the key muscle protein, dystrophin.
Without dystrophin, cells easily become
damaged and die, resulting in heart and
breathing failure.
What is CureDuchenne?
CureDuchenne is a nonprofit that raises awareness and
funds research to find a cure for Duchenne.
POSSIBLE LEARNING AND
COGNITIVE DIFFICULTIES
CUREDUCHENNE’S COMPREHENSIVE
RESEARCH STRATEGY:
treatments to reduce disease severity
Slow progress of the disease
Affected boys
usually are diagnosed
before age 5 ...
DECREASED HEART FUNCTION
CARDIOMYOPATHY
LEADS TO HEART FAILURE
drug repositioning
New use of existing drugs
protein replacement
Restore dystrophin and complementary proteins
CUREDUCHENNE’S IMPACT
WEAKENS DIAPHRAGM
REQUIRES VENTILATOR IN
TEENS
LEADS TO PNEUMONIA
... confined to
wheelchairs
by age 12 ...
LOSS OF MUSCLE MASS
WEAKNESS
INFLAMMATION
FIBROSIS
....and most
don’t survive
their mid-20s.
Number of research projects advanced
into human clinical trials with
CureDuchenne’s support.
7
2014
Year expected to see first drugs approved.
Two drug development programs supported
by CureDuchenne will be the first to seek
approval for treatment of Duchenne.
$100 MILLION
Amount of money leveraged from
government agencies and pharmaceutical
companies to fund research leading to a cure.
100 MILLION
Number of people reached through
awareness campaign.
Based on current scientific knowledge, Duchenne
could become treatable for this generation of boys.
BRITTLE AND WEAK
We just need the funding now. Help us
CureDuchenne Now. www.cureduchenne.org

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