Myotonic dystrophy type 1 is a rare, dominantly inherited, progressive, disabling, neuromuscular disease that leads to decreased life expectancy and has no approved therapies. The disease is caused by ...

WVE-N531 showed significant improvements in functional measures and muscle health in DMD patients during the phase 2 FORWARD-53 trial. Sustained exon skipping, dystrophin restoration, and improved ...

Scientists have identified previously unreported genes which appear to play a key role in the muscle ageing process. It is hoped that the findings from the Nottingham Trent University study could be ...

Many patients are given a different diagnosis initially, including lupus erythematous and undifferentiated connective tissue disease. It is of paramount importance to determine the organ (i.e. skin, ...

Duchenne muscular dystrophy (DMD) and Becker muscular dystrophy (BMD) are caused by mutations in the DMD gene, which reduces the amount of dystrophin, a protein that protects muscle fibers. DMD ...