Sarepta has stopped developing SRP-5051, its exon 51-skipping therapy for Duchenne MD, due to long-term safety concerns and ...

Specific mutations in the DMD gene influence how long patients retain the ability to walk, even when treated with ...

I have three teenage sons — Max, 18, Rowen, 15, and Charlie, 13 — who have Duchenne muscular dystrophy (DMD). They have met several sweet milestones over the years, and I love celebrating them. When ...