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 ...

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 ...

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

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 ...

People living with a chronic illness, including me, commonly explore how we deal with grief, which is the subject of much theory and research. I’ve thought deeply about how living with limb-girdle ...