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Sarepta Therapeutics (SRPT) said Friday that three patients born with a rare muscle-wasting disease called limb-girdle muscular dystrophy showed improvements in strength and function when measured nine months after a one-time infusion of an experimental gene therapy.

The encouraging limb-girdle gene therapy data are a welcome dose of good news for Sarepta, which has seen its problems mount and its stock price fall since the Food and Drug Administration rejected a Duchenne muscular dystrophy drug in August. Sarepta shares were up 6% Friday.


However, as with all gene therapies, Sarepta still needs to confirm the durable benefit of its limb-girdle therapy, which means treating more patients and following them for a longer period of time.

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