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

Columnist Betty Vertin wishes she could take away the pain over the milestones in the teenage years her sons with DMD will miss.

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

A new patient group is being enrolled in a Phase 1/2 trial of delpacibart braxlosiran (del-brax), an experimental and potentially disease-modifying therapy for facioscapulohumeral muscular dystrophy ...