U.S. FDA approved first gene therapy for treatment of patients with Duchenne Muscular Dystrophy

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On Jun. 22, 2023, the U.S. Food and Drug Administration (FDA) approved Sarepta Therapeutics’s Elevidys, the first gene therapy for the treatment of pediatric patients 4 through 5 years of age with Duchenne muscular dystrophy (DMD) with a confirmed mutation in the DMD gene who do not have a pre-existing medical reason preventing treatment with this therapy.

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Source: U.S. Food and Drug Administration
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