Patient advocate Austin Leclaire, who died Feb. 1, played a pivotal role in spurring the development of the first medicines ...

Some muscular dystrophies are mild. Others are more serious and can cause life-threatening muscle weakness. Most children with Duchenne ... years to progress. This form of muscular dystrophy ...

Duchenne muscular dystrophy (DMD) is a genetic condition ... it can help stabilize the progression of symptoms and may help improve strength and endurance. Elevidys involves modifying a virus ...