FDA Approval for Duchenne Muscular Dystrophy Gene Therapy

Published 2023-07-23

FDA recently approved the first gene therapy for Duchenne Muscular Dystrophy (DMD) for pediatric patients between 4-5 years of age. The drug, Elevidys, made by Sarepta Therapeutics is a adeno-associated virus vector-based gene therapy targeting the DMD gene. 

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