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

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

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 ...

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 ...

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 ...

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

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.