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 ...

Recent studies have defined a group of muscular dystrophies, now termed the dystroglycanopathies, as novel disorders of glycosylation. These conditions include Walker–Warburg syndrome, ...

Early screening for Duchenne muscular dystrophy can help you get early treatments and referrals for your child to help prevent further loss of muscle tissue. Duchenne muscular dystrophy (DMD) is a ...

Becker muscular dystrophy (BMD) is a genetic disorder that causes progressive muscle degeneration and weakness. This can cause a person to have reduced mobility such as problems walking. BMD can make ...

Shares of Cambridge-based Sarepta rose about 35 percent in trading Wednesday after the company presented preliminary data on ...

The investigational gene therapy SGT-003 is showing early signs of safety and efficacy for Duchenne muscular dystrophy in a clinical trial.

The authors of this report stress the importance of awareness of COVID-19–related complications in young patients who have Duchenne muscular dystrophy, paying particular attention to a potential ...