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.

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 ...

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 improvement in muscles.

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

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

Capricor plans to resubmit its application for the approval of deramiocel for DMD after the FDA declined to accept the original proposal.

Columnist Betty Vertin embraces July's slower pace, a much-needed respite from caregiving demands for her family of nine.