Sarepta's gene therapy fails primary goal in rare muscular dystrophy trial

 Sarepta Therapeutics' gene therapy to treat Duchenne muscular dystrophy (DMD) failed to meet the main goal of a late-stage trial, the company reported after the bell on Monday.

The therapy failed to meet statistical significance in improving patient clinical outcomes such as their ability to walk and stand when compared to placebo-treated patients at 52 weeks.

DMD is an inherited progressive muscle-wasting disorder that almost always affects young boys in the ages of 4 through 7 years.

The U.S. Food and Drug administration in June approved the treatment for children aged between 4 and 5 years who can walk. Sarepta was initially seeking approval for all DMD patients who can walk. 

https://finance.yahoo.com/news/1-sareptas-gene-therapy-fails-202827071.html