Agamree, an approved steroid treatment for Duchenne MD, will be sold in Turkey by GEN, per an agreement with Santhera.

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 ...

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

The first patient has been dosed in a clinical trial testing Sardocor's one-time gene therapy for cardiomyopathy associated with Duchenne MD.

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.