SRP-9001

SRP-9001 is a gene therapy developed by Sarepta Therapeutics to treat Duchenne muscular dystrophy (DMD), a genetic disorder causing progressive muscle weakness. It works by delivering a modified version of the gene that produces a functional form of dystrophin, a protein essential for muscle health that is missing in DMD patients. This therapy aims to help the body produce some dystrophin again, potentially improving muscle strength and slowing disease progression. It is administered via an infusion and represents a significant advancement in addressing the underlying cause of DMD.