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FDA OKs Elevidys As First Gene Therapy To Treat Duchenne Muscular Dystrophy

FDA OKs Elevidys As First Gene Therapy To Treat Duchenne Muscular Dystrophy

Sarepta Therapeutics, Inc. announced Friday that the U.S. Food and Drug Administration or FDA has accelerated approval of ELEVIDYS (delandistrogene moxeparvovec-rokl) for the treatment of ambulatory pediatric patients aged 4 through 5 years with Duchenne muscular dystrophy (DMD) with a confirmed mutation in the DMD gene.
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Sarepta Therapeutics Says FDA Lifts Clinical Hold On SRP-5051 To Treat Duchenne Muscular Dystrophy

Sarepta Therapeutics Says FDA Lifts Clinical Hold On SRP-5051 To Treat Duchenne Muscular Dystrophy

Genetic medicine maker Sarepta Therapeutics, Inc. (SRPT) announced Tuesday that the U.S. Food and Drug Administration (FDA) has removed the clinical hold on SRP-5051 (vesleteplirsen), the Company's investigational, next-generation peptide-conjugated phosphorodiamidate morpholino oligomer (PPMO) to treat patients with Duchenne muscular dystrophy who are amenable to exon 51 skipping.
RTTNews | Pred 784 dňami