Preliminary data are expected this year for a Phase 1/2 clinical trial testing the investigational therapy SRP-1003 in DM1.

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

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

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 ...

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

Columnist Robin Stemple, his brothers, and more enjoy a long-in-the works family music celebration at his mother's nursing home.

In both good times and bad, the importance of human connection cannot be understated, columnist Patrick Moeschen writes.

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

The FDA asked Sarepta to pause all U.S. shipments of the DMD gene therapy Elevidys, and the company has agreed after initially refusing.

Del-zota was granted FDA breakthrough therapy designation for treating DMD, and its developer is on track to seek its regulatory approval.