Accelerating access to Duchenne drug candidates
CureDuchenne is now funding $7 million Prosensa exon skipping research to help get experimental medicines to patients with Duchenne Muscular Dystrophy more quickly.
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.
The Latest from the Founder
CureDuchenne is delighted to share that Prosensa has begun re-dosing patients. See below for today’s announcement. We are happy that CureDuchenne funding is helping to move Prosensa’s exon skipping programs forward. Our $7 million collaboration with Prosensa is helping to accelerate access to exon skipping drugs for several mutations for patients who so desperately need it. Read more »Continue reading