A protein called MYOD has long been known to be able to turn on genes, but a recent study has found that it also can turn off ...

Enrollment is now ongoing in a first-in-human clinical trial testing EPI-321, a therapy designed to target the underlying ...

Columnist Shalom Lin and his partner, Amanda — both disabled artists — find that their challenges are exactly what can drive ...

The FDA has granted breakthrough therapy designation to DYNE-251 for the treatment of DMD in patients amenable to exon 51 ...

Columnist Betty Vertin, whose days often feel overloaded, describes how it feels when her life as a caregiver is recognized and affirmed.

A mouse model study of Precision BioSciences’ gene-editing therapy PBGENE-DMD found it produced long-term functional ...

Share this article: An experimental gene editing therapy for Duchenne muscular dystrophy (DMD) being developed by Precision Biosciences has received orphan drug status from the U.S. Food and Drug ...

Sarepta will soon resume shipping Elevidys for ambulatory Duchenne muscular dystrophy patients after a review of safety data ...

The FDA has granted rare pediatric disease designation to PBGENE-DMD, an experimental gene-editing therapy for Duchenne muscular dystrophy.

Decker was given the 2025 MDA Legacy Award, recognizing his efforts to advance gene therapy, scientific research, and the patients' voice.

A young man with Duchenne MD and treated with Elevidys has died of acute liver failure, Sarepta reports; it's working with health officials.