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Friday, December 12, 2025

Second Patient Dies After Gene Therapy for Duchenne Muscular Dystrophy: Company

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Sarepta, the company that makes the drug, is pausing it for non-ambulatory patients.

A second patient has died after receiving the gene therapy Elevidys, which was approved by the Food and Drug Administration (FDA) over concerns of staffers, according to the company that makes it.

The 15-year-old boy who died, and the 16-year-old boy whose death was reported earlier in the year, had both received Elevidys, the only gene therapy approved by the FDA for the treatment of a rare form of muscular dystrophy called Duchenne muscular dystrophy (DMD), Sarepta officials said in a call on June 16.

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