Muscular Dystrophy (MD) is a genetic condition causing progressive muscle weakness and complications in vital organs. Early ...

Top-line Phase 3 trial data show ambulatory boys with Duchenne continued with motor improvements two years after a single ...

While the last decade has brought considerable progress for patients with DMD, substantial unmet need remains. Several ...

In 1987, the muscle protein associated with this gene was named dystrophin. Duchenne muscular dystrophy occurs when that gene fails to make dystrophin. Becker muscular dystrophy occurs when a ...

The gene therapy improved motor functions in children with Duchenne muscular dystrophy two years after treatment ...

Sarepta's EMBARK study confirms Elevidys offers sustained Duchenne treatment benefits, including motor function improvement ...

Biophysical characterization of the dystrophin C-terminal domain: Dystrophin interacts differentially with dystrobrevin isoforms. Journal of Biological Chemistry , 2024; 300 (12): 108002 DOI: 10. ...

Expert diagnostic services and treatment are provided for patients with common and rare disorders of the muscular system, including muscular dystrophies. Myopathic (myo = muscle, pathy = disease) ...

Although the drug increases dystrophin production, which would predict improvement in muscle function, this has not yet been shown. The first gene therapy to be approved for DMD, delandistrogene ...