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

Muscular dystrophy News, a property within the Bionews network of rare disease websites, is a community dedicated to providing resources for people living with muscular dystrophy and their care ...

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

Elevidys (delandistrogene moxeparvovec-rokl), previously called SRP-9001, is a onetime gene therapy approved for certain patients with DMD.

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 ...

Columnist Patrick Moeschen emphasizes the importance of making advance directives and shares some tips for how to get started ...

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

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

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 ...

Exon skipping is a treatment for people with Duchenne muscular dystrophy that may make the effects of the genetic disease less severe.