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Sarepta Therapeutics on Thursday announced mixed results from the first randomized clinical trial of its gene therapy for Duchenne muscular dystrophy, raising questions about the path forward for the one-time, potentially curative treatment.

A single infusion of the treatment, called SRP-9001, produced large increases in a crucial muscle protein typically missing in children born with Duchenne. But the increases failed to coincide with statistically significant improvements in muscle function for all patients after one year.

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