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.

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

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 ...

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

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 ...

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

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

What if the risk vs. reward choice focused on your medical care or treatment options? How do you weigh risk vs. reward if your life is at stake, and how do you weigh your own morals, ethics, and ...

Ifetroban improved heart function and reduced cardiac damage biomarkers in people with DMD in a Phase 2 clinical trial.