The first drug to treat the underlying cause of Duchenne muscular dystrophy, Exondys (eteplirsen) developed by Sarepta Therapeutics, has secured marketing approval from the U.S. Food and Drug Administration. Duchenne muscular dystrophy is a severe type of muscular dystrophy which generally begins around age four in boys and progresses rapidly.

www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm521263.htm