STAT+: Sarepta’s Duchenne gene therapy improves muscle function but debate lingers

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.

Continue to STAT+ to read the full story…