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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 way forward for one-time and potentially curative treatment.
A single infusion of the treatment, called SRP-9001, produced large increases of a crucial muscle protein generally absent in children born with Duchenne. But the increases did not coincide with statistically significant improvements in muscle function for all patients after one year.
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