Patient stories

Children’s Hospital of Philadelphia now offers the first-ever gene therapy treatment for Duchenne Muscular Dystrophy (DMD) to patients like 6-year-old Cash. This breakthrough treatment slows the progression of symptoms and can improve strength and endurance for patients with DMD.

Cooper was one of the first at CHOP to receive a new FDA-approved infusion therapy for Duchenne muscular dystrophy, which is given to him at home. Results are promising.