Columnist Patrick Moeschen, who lives with limb-girdle muscular dystrophy, explains why controlling what he can is so ...

Regenxbio hopes to use the study's results to ask the U.S. Food and Drug Administration to grant accelerated approval to the ...

As a caregiver for three sons with DMD, columnist Betty Vertin clung to her many responsibilities. This year she's learned to let go a bit.

Columnist Shalom Lim tells an international conference about his experiences with inclusion during his education in Singapore ...

The FDA has given the green light to a clinical trial testing ATA-200, a gene therapy for limb-girdle muscular dystrophy ...

Sarepta has stopped developing SRP-5051, its exon 51-skipping therapy for Duchenne MD, due to long-term safety concerns and ...

Regenxbio has initiated the pivotal phase of a clinical trial testing its experimental gene therapy RGX-202 in boys with Duchenne muscular dystrophy (DMD). If the results are positive, they could ...

Shalom Lim is a University of Liverpool graduate, having completed an honors bachelor’s degree in criminology and security in July 2021. Born with Duchenne muscular dystrophy and diagnosed at 4 months ...

A new technology called StitchR effectively restored the production of large muscle proteins dystrophin and dysferlin in mouse models of Duchenne muscular dystrophy (DMD) and limb-girdle muscular ...

Despite positive trial data, Sarepta Therapeutics has decided to stop the clinical development of SRP-5051 (vesleteplirsen), an exon 51-skipping therapy for some people with Duchenne muscular ...