Sarepta Therapeutics (NasdaqGS:SRPT) recently announced that its SRP-9003 gene therapy received a platform technology designation from the FDA, and updated safety protocols for ELEVIDYS in the UK.

Sarepta suspends Elevidys use in non-ambulatory Duchenne patients after two liver failure deaths, triggering FDA review and ...

Another gene therapy death of a Duchenne patient has some parents and doctors criticizing the FDA's actions, even as other ...