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Duchenne gene therapy halted after three deaths and company u turn

bmj · 2025-07-23 · canonical JSON source

5 visible annotations · policy: published · automated confidence ≥ 75.00%

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The biotechnology company Sarepta Therapeutics has decided to suspend entirely one of its treatments for Duchenne muscular dystrophy (DMD) just days after it refused a request from the US Food and Drug Administration (FDA) to pull the drug from the market.