Help Tyler Armstrong Climb to CureDuchenne.
Tyler is not just trying to set a world record; his goal is to raise awareness and funds to cure Duchenne muscular dystrophy, the most common and lethal muscle disease found in children. Duchenne afflicts approximately 300,000 boys worldwide.
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.
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.
Help Advance Research to Treat Heart Disease
CureDuchenne Ventures LLC is investing $1 million in Capricor Therapeutics to advance promising research to treat disease associated with Duchenne muscular dystrophy.
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.
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!
The Latest from the Founder
Duchenne Community: Let Your Voice Be Heard! August 27, 2015
This week the FDA accepted Sarepta Therapeutics’ New Drug Application for eteplirsen with priority review. This follows on the June FDA’s acceptance of BioMarin’s NDA for drisapersen, also with priority review. The next step for both of these drugs is an FDA Advisory Committee meeting in November where both companies will demonstrate why these Read more »Continue reading