Sarepta Therapeutics said Monday that its gene therapy improved muscle function for a group of patients with Duchenne muscular dystrophy — updated results from a mid-stage study the company believes ameliorates an initial negative outcome last year and might support accelerated approval.
But the magnitude of the benefit shown by Sarepta’s one-time treatment was lower than some investors hoped to see. That concern, coupled with weaker financial guidance for the year, caused Sarepta’s stock price to drop.
Duchenne, a disease that almost always affects boys, is caused by an inherited mutation in a gene that produces dystrophin, a protein required to make muscles function properly. Without dystrophin, muscles break down over time, which is why Duchenne patients gradually lose their ability to walk and breathe. The disease is fatal, often by the time boys are in their late teens or early 20s.
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