The FDA has given the green light to a clinical trial testing ATA-200, a gene therapy for limb-girdle muscular dystrophy ...

Despite positive trial data, Sarepta Therapeutics has decided to stop the clinical development of SRP-5051 (vesleteplirsen), ...

Columnist Robin Stemple says he chooses to focus on what FSHD progression hasn't taken from him. The small victories matter, ...

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

Despite positive trial data, Sarepta Therapeutics has decided to stop the clinical development of SRP-5051 (vesleteplirsen), an exon 51-skipping therapy for some people with Duchenne muscular ...

Betty Vertin is a mother and writer living in rural Hastings, Nebraska, with her husband and seven children. Betty is a caregiver to three sons, Max, Rowen, and Charlie. Her oldest was diagnosed 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 ...

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 ...