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FDA OKs trial of SRP-9005 gene therapy for LGMD type 2C

Summary by Muscular Dystrophy News
The U.S. Food and Drug Administration (FDA) has cleared Sarepta Therapeutics to start dosing and screening in a first clinical trial testing the gene therapy SRP-9005 in people with limb-girdle muscular dystrophy type 2C (LGMD2C). Sarepta said advancement continues for SRP-9003 and SRP-9004, two gene therapies already in clinical testing for other types of LGMD. “There are no disease-modifying treatments approved for patients with any subtype of…
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Muscular Dystrophy News broke the news in on Thursday, April 17, 2025.
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