Bay Ridge family lauds approval of first-ever drug for Duchenne Muscular Dystrophy
One local family is celebrating the U.S. Food and Drug Administration (FDA)’s recent approval of Exondys 51 (also known as Eteplirsen), the first drug approved to treat patients with Duchenne Muscular Dystrophy.
The disease – known for short as DMD – is a recessive X-lined form of muscular dystrophy that affects one in every 3,500 to 5,000 boys, including Bay Ridge’s own Pietro Scarso, a seven-year-old boy who was diagnosed at just three years old.
Currently, the second grader is in his 62nd week of trials for the drug which… read full article >>
By Meaghan McGoldrick firstname.lastname@example.org
Wednesday, September 21, 2016 3:13 PM