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 the one-off, potentially curative treatment.
A single infusion of the treatment, called SRP-9001, caused large increases in an important muscle protein that is usually lacking in children born with Duchenne. However, the increases could not coincide with statistically significant improvements in muscle function for all patients after one year.
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