Sarepta heading toward pivotal trial of first limb-girdle muscular dystrophy gene therapy

A new readout clears the path for Sarepta to move ahead next year with a pivotal study of its gene therapy for limb-girdle muscular dystrophy type 2E, one of several LGMD subtypes the company is seeking to treat.

The data also indirectly add to the body of evidence for the safety of a microdystrophin-targeting gene therapy for Duchenne muscular dystrophy from Sarepta Therapeutics Inc. (NASDAQ:SRPT), which uses the same vector and promoter as the LGMD therapy and is delivered at the same dose...