STAT Plus: Sarepta gene therapy improves muscle function for patients with rare limb-girdle disease
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.
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