welcome to DuchenneXchange

- a positively charged Duchenne muscular dystrophy community.
  • join today!

Sarepta Therapeutics Completes Submission of New Drug Application Seeking Approval of Golodirsen (SRP-4053) in Patients with Duchenne Muscular Dystrophy Amenable to Skipping Exon 53


Sarepta Therapeutics, Inc., a leader in precision genetic medicine for rare diseases, announced today that it has completed the submission of its rolling New Drug Application (NDA) seeking accelerated approval for golodirsen (SRP-4053), a phosphordiamidate morpholino oligimer engineered to treat those patients with Duchenne muscular dystrophy who have genetic mutations subject to skipping exon 53 of the Duchenne gene. Duchenne is a fatal genetic neuromuscular disorder affecting an estimated one in approximately every 3,500 – 5,000 males born worldwide.

Sarepta Therapeutics Completes Submission Of New Drug Application Seeking Approval Of Golodirsen (SRP-4053) In Patients With Duchenne Muscular Dystrophy Amenable To Skipping Exon 53