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Sarepta Duchenne gene therapy wins broader use from FDA

The Food and Drug Administration has substantially loosened limits on the first gene therapy for Duchenne muscular dystrophy in a decision that could greatly expand its use even as questions remain about its effectiveness.

The agency on Thursday made the therapy, called Elevidys and sold by biotechnology company Sarepta Therapeutics, available to people with Duchenne who are at least four years of age and have mutations in a specific gene, regardless of whether they can still walk.

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