Top FDA Official Overrides Agency Staff to Approve Sarepta Gene Therapy
Sarepta Therapeutics gene therapy, Elevidys, has received traditional FDA approval after failing its confirmatory test, expanding use to a wider group of Duchenne muscular dystrophy patients. The therapy works by producing a smaller version of dystrophin, a protein key to muscle function. The therapy was initially approved based on Phase 2 data showing micro-dystrophin production, […]