Data from the EMBARK trial of delandistrogene moxeparvovec in patients with Duchenne muscular dystrophy (DMD) show that ...

Children with SMA given the gene therapy Zolgensma in infancy are maintaining motor milestones after up to 10 years, new ...

The young man died of acute liver injury, a known side effect, Sarepta said in a statement. But the company said the ...

Myotonic dystrophy type 1 (DM1) is the most common adult-onset form of muscular dystrophy and a condition that severely ...

The rapid development of gene therapy options for treating neuromuscular diseases has created new therapeutic options but ...