The U.S. Food and Drug Administration on Thursday approved the drug Elevidys, the first gene therapy for the treatment of children with Duchenne muscular dystrophy (DMD).
The groundbreaking treatment will not be cheap: Drugmaker Sarepta Therapeutics Inc. said it would charge $3.2 million for the one-time IV treatment, the Associated Press reported. Like most medicines in the Unit...
An experimental gene therapy for Duchenne muscular dystrophy shows promise, a small study suggests.
The severe form of muscular dystrophy -- which affects about one in 3,500 males born each year in the United States -- causes muscles to progressively weaken and lose the ability to regenerate after an injury.
Muscle tissue is eventually replaced by fat and collagen. Many children wit...
Folks who get treatment from a stem cell clinic could be spending their money on what amounts to snake oil, a new study warns.
Doctors administering stem cells might have no expertise in the condition they're trying to treat, and the cells themselves might be derived from questionable or discredited sources -- if the treatment contains any stem cells at all.