Sarepta’s latest tragedy calls for FDA leadership, industry collaboration on AAV liver toxicity

The tragic death of a Duchenne muscular dystrophy patient in a trial of Sarepta’s Elevidys gene therapy presents FDA’s leadership with a test of its competence and an opportunity to demonstrate leadership.

Sarepta and other companies that have developed or are testing AAV gene therapies that could pose similar safety risks have a responsibility to share data with the patient community, FDA and each other. ...