Sarepta Therapeutics suffered another major setback on Friday as Europe's drug regulator decided not to recommend the ...

The U.S. Food and Drug Administration said on Friday it was investigating the death of an eight-year-old boy who received ...

Separately, Sareptea said last week that it would pause the development of most of its experimental gene therapies for a different type of muscular dystrophy. The stoppage came after one of the ...

As gene therapy maker Sarepta Therapeutics tangles with FDA over its Duchenne treatment, patients and their families are ...