NEWPORT BEACH, Calif.--(BUSINESS WIRE)-- CureDuchenne, a nonprofit that raises awareness and funds research to find a cure for Duchenne muscular dystrophy, is pleased by todays announcement by Sarepta Therapeutics (formerly AVI BioPharma) that its exon-skipping compound, eteplirsen, achieved significant clinical benefit after 48 weeks of treatment in a Phase IIb study in Duchenne muscular dystrophy. CureDuchenne, along with Children National Medical Center in Washington, D.C., and the Foundation to Eradicate Duchenne, provided funding in 2010 for this research to progress into human clinical trials. The eteplirsen drug helped increase the production of dystrophin, which is lacking due to a defect on the dystrophin gene that causes Duchenne, and slowed the progression of the disease, as measured by the 6-minute walk test. This is a milestone for the company and patients alike. The result will pave the way for an end of phase II meeting between the company and the FDA to discuss and agree to the plan for a phase III registration trial. Sarepta will scale up the manufacturing to accommodate a larger trial. This is an important day for Duchenne, said Chris Garabedian, CEO of Sarepta Therapeutics. We are grateful to CureDuchenne, and other organizations, for their support and … Continue reading
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