An experimental RNA therapy from Avidity Biosciences has early clinical trial results showing it reduced by half the expression of a gene at the root of a rare, inherited form of muscular dystrophy ...

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 ...

One of the first symptoms of LGMD is typically a weakening of the muscles in the hip and shoulder girdles. The hip girdle is a ring-like bone structure in the pelvic area, consisting of the pelvis, ...

Delandistrogene moxeparvovec, a gene therapy approved for the treatment of Duchenne muscular dystrophy (DMD), was found tolerable and showed signs of efficacy in a real-world cohort. Delandistrogene ...

Muscular dystrophy, known as DMD, is a rare and fatal genetic disease that primarily affects boys, slowly robbing them of their ability to walk, breathe and live independently. Until now, there have ...

In boys with DMD, executive skills such as self-control begin to lag around age 8, but show signs of partial catch-up by 14, ...

HOUSTON – There’s a new type of therapy that can help kids with Duchenne muscular dystrophy. Duchenne muscular dystrophy (DMD) affects one in 3,600 male births, and it can lead to immobility, and ...

Decreased strain and rotation were hypothesized to be associated with the presence and extent of left ventricular myocardial fibrosis in Duchenne muscular dystrophy. Investigators recently ...