Sarepta, days out from critical FDA meeting, lists questions expected at gene therapy AdComm

The clock is ticking down to the FDA advisory committee meeting about Sarepta Therapeutics’ Duchenne muscular dystrophy (DMD) gene therapy SRP-9001. With days to go, Sarepta has set out what is expected to be asked at the meeting—and made the case that it can allay concerns about its confirmatory trial.