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 ...

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

Sarepta will soon resume shipping Elevidys for ambulatory Duchenne muscular dystrophy patients after a review of safety data ...

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

A public workshop that columnist Shalom Lim and his girlfriend, Amanda, organized aimed to foster connection among people ...

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.

Dealing with pain has become increasingly challenging for columnist Robin Stemple, who struggles to find relief from the hurt ...

DMD treatment KER-065 showed a good safety profile in a Phase 1 clinical trial with no serious side effects seen in healthy volunteers.

Decker was given the 2025 MDA Legacy Award, recognizing his efforts to advance gene therapy, scientific research, and the patients' voice.