Sarepta Receives Historic, Accelerated Approval from FDA

PharmApprove congratulates Sarepta Therapeutics on receiving accelerated approval for the first drug to treat Duchenne Muscular Dystrophy (DMD) in the US.  EXONDYS 51 has been approved for patients who have a confirmed mutation in the DMD gene that is amenable to exon 51 skipping.

This is an historic moment for Duchenne boys, their families and the larger rare disease community.