SRPT - Sarepta/Roche's Closely Watched Duchenne Gene Therapy Faces More FDA Scrutiny - Stock Tanks | Benzinga
Sarepta Therapeutics Inc (NASDAQ: SRPT) released topline results from EMBARK (Study SRP-9001-301) Phase 3 study of Elevidys (delandistrogene moxeparvovec-rokl) in ambulatory boys (those who can walk) with Duchenne muscular dystrophy aged 4-7 years.
In June, the FDA granted accelerated approval to gene therapy, Elevidys, for Duchenne muscular dystrophy in ambulatory pediatric patients aged 4 through 5 years with a confirmed mutation in the DMD gene.
Elevidys-treated patients improved by 2.6 points on their ...