Skeletal muscle is a dynamic and highly adaptable tissue responsible for voluntary movement, posture and metabolic regulation. Its unique architecture, involving myofibres, satellite cells and a ...
The muscular dystrophies are commonly associated with cardiovascular complications, including cardiomyopathy and cardiac arrhythmias. These complications are caused by intrinsic defects in ...
Congenital muscular dystrophy (CMD) is a disease that affects certain muscles. Individuals with CMD may experience symptoms such as muscle weakness and joint problems. “Congenital” means present since ...
Facioscapulohumeral muscular dystrophy (FSHD) is a genetic condition that can lead to the slow deterioration of muscles, predominantly in the face, shoulder blades, and upper arms. Facioscapulohumeral ...
Duchenne therapy may be evolving from slowing disease to reversing it—with RNA approaches offering the prospect of restoring function. Ongoing research will determine if these advances can deliver ...
A new gene therapy treatment for Duchenne muscular dystrophy (DMD) shows promise of not only arresting the decline of the muscles of those affected by this inherited genetic disease, but perhaps, in ...
We hypothesize that Duchenne muscular dystrophy and autism spectrum disorder/pervasive developmental disorder co-occur with a greater than random frequency. In this study, we set out to reject the ...
Delandistrogene moxeparvovec demonstrated a manageable safety profile across clinical trials for Duchenne muscular dystrophy (DMD), with most adverse events emerging within 90 days of infusion. Barry ...