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Sarepta Therapeutics and Nationwide Children’s Hospital Announce FDA Clearance of IND for Micro-Dystrophin Gene Therapy Program for the Treatment of Duchenne Muscular Dystrophy

CAMBRIDGE, Mass., Nov. 06, 2017 (GLOBE NEWSWIRE) — Sarepta Therapeutics, Inc. (NASDAQ:SRPT), a commercial-stage biopharmaceutical company focused on the discovery and development of precision genetic medicines to treat rare neuromuscular diseases, today announced that Nationwide Children’s Hospital has received clearance from the U.S. Food and Drug Administration (FDA) for its Investigational New Drug (IND) application […]


Jerry Mendell, in PPMD Webinar, Details Plans for Possible Trial of Duchenne Gene Therapy

In a recent webinar, Dr. Jerry Mendell with Nationwide Children’s Hospital rolled out plans for a Phase 1/2a clinical trial of a new gene therapy for Duchenne’s muscular dystrophy (DMD). Final go-ahead for the trial awaits U.S. Food and Drug Administration (FDA) approval of an Investigational New Drug (IND) application1 made for the therapy and its testing. But Mendell is […]


MicroRNA-29 overexpression by adeno-associated virus suppresses fibrosis and restores muscle function in combination with micro-dystrophin

Our overall goals include developing meaningful treatment for DMD boys at any stage of disease and one of the challenges to treat DMD is the accumulation of fibrosis or scar tissue as muscle loss progresses.  We have previously demonstrated a way to replace the missing dystrophin gene with a miniature dystrophin using gene therapy.  We […]


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