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 ...

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

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

The FDA has granted breakthrough therapy designation to DYNE-251 for the treatment of DMD in patients amenable to exon 51 ...

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.

A mouse model study of Precision BioSciences’ gene-editing therapy PBGENE-DMD found it produced long-term functional improvement in muscles.

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

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