It’s far too early to say whether gene therapy can treat Duchenne muscular dystrophy, a deadly genetic disease. But the first clinical data have arrived, with Sarepta Therapeutics today reporting encouraging preliminary results from three patients. Sarepta (NASDAQ: SRPT), of Cambridge, MA, is conducting an early-stage study of its experimental treatment known as AAVrh74.MHCK7.micro-Dystrophin. The […]
Original Article: Sarepta’s Data Are First in Rush for Duchenne Gene Therapy