Duplication Mutation Therapy for Duchenne
CureDuchenne has provided the lead funding for Dr. Flanigan's duplication research and is committed to helping bring this therapy into trials. We need your support in raising $710,000 to continue this important research.
Moving research into practice
CureDuchenne’s accelerated push to move research from the lab into clinical trials could halt the progression of the disease and save the lives of thousands of Duchenne boys.
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 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.
Champions to CureDuchenne is a prestigious, annual charity wine auction benefiting CureDuchenne. We hope you’ll join us as select group of wine connoisseurs, vintners, community and business leaders come together for a fun and memorable evening which will include a wine tasting, custom designed dinner from our celebrity chef, and a live and silent auction.Register Now!
The Latest from the Founder
Pfizer Inc. (NYSE:PFE) announced today enrollment of the first patient in a multicenter Phase II clinical trial of the investigational compound PF-06252616 in boys with Duchenne muscular dystrophy (DMD), a genetic disorder characterized by progressive muscle degeneration and weakness. PF-06252616 is an experimental, infused, anti-myostatin monoclonal antibody. Myostatin is a naturally occurring protein in muscles Read more »Continue reading