sArepta Therapeutics announced on Thursday the mixed results of the first randomized clinical trial of its gene therapy for Duchenne muscular dystrophy, raising questions about the way forward for the unique and potentially curative treatment.
A single infusion of the treatment, called SRP-9001, produced large increases in a crucial muscle protein that is often lacking 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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