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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.


Sarepta said it was “very disappointed” in the outcome of the study overall but expressed confidence that its gene therapy was effective, demonstrated by significant improvements in muscle function reported by younger children enrolled in the study.

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