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.

Doctors can use several tests to diagnose muscular dystrophy (MD). A correct diagnosis gives patients an understanding of how their disease is likely to progress. It also helps doctors do a better job ...

There is no cure for any type of muscular dystrophy (MD), but various medications and therapies can help manage symptoms or slow the progression of the disease. Many therapies are specific to one type ...

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