Regenxbio hopes to use the study's results to ask the U.S. Food and Drug Administration to grant accelerated approval to the ...

Columnist Shalom Lim tells an international conference about his experiences with inclusion during his education in Singapore ...

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 ...

Life feels busy, which is no surprise to me. My husband, Jason, and I share seven children: Lexi, 23; Max, 19; Chance, 17; Rowen, 15; Charlie, 13; Mary, 10; and Callie, 2. Max, Rowen, and Charlie live ...

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

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

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 ...

Columnist Betty Vertin hopes her family's experience with kidney stones might help you be better prepared while living with DMD.

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 ...

A new technology called StitchR effectively restored the production of large muscle proteins dystrophin and dysferlin in mouse models of Duchenne muscular dystrophy (DMD) and limb-girdle muscular ...