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