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CureDuchenne is proud to announce Small Luxury Hotels of the World as their national hotel sponsor.
Together, we can CureDuchenne
Duchenne muscular dystrophy is a devastating muscle disease in children, affecting one in every 3,500 male births. Historically, Duchenne boys have not lived beyond their mid-20s. Those that do are faced with progressively weaker muscles and pulmonary and cardiac failure. The potential now exists, however, to make a difference in their quality of life.
Duchenne families have hope
Hear what Duchenne boys, their parents, and researchers have to say about the experience of living with Duchenne muscular dystrophy.
The eDystrophin database contains information from Duchenne and Becker muscular dystrophy patients searchable by mutation (deletions, duplications and substitutions).
Duchenne is a progressive, muscle-wasting disease.
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.
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What is CureDuchenne?
CureDuchenne is a national nonprofit that raises awareness and funds to find a cure for Duchenne muscular dystrophy. The funds we raise support the most promising research programs aimed at treating and curing the disease. For the first time ever there is hope to find a cure for this destructive disease affecting thousands of boys and their families.
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Exon skipping drugs will be used to transform Duchenne muscular dystrophy into a much milder disease as seen in Becker muscular dystrophy patients. eDystrophin is an online database that provides information about inframe mutations typically seen in patients with milder forms of the disease. The database has been constructed using patient records and is easy to use and searchable by mutation type or clinical phenotype.
Join Ryan Getzlaf, captain of the Anaheim Ducks, for the Getzlaf Golf Shootout – a two-day charity event, including a reception and a golf tournament – that brings together athletes, celebrities and community leaders, all teaming up in support of CureDuchenne.Register Now!
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Faster Cures Innovator Spotlight Features Cure Duchenne July 24, 2014
FasterCures features a Q&A with Debra Miller, founder and CEO of CureDuchenne, in their Innovator Spotlight. FasterCures, a center of the Milken Institute, is an action tank driven by a singular goal – to save lives by speeding up and improving the medical research system. The Q&A highlights CureDuchenne’s successful venture philanthropy model. It focuses Read more »Continue reading