Monday, September 19, 2016

FDA grants accelerated approval to first drug for Duchenne muscular dystrophy - FDA Press Releases

The U.S. Food and Drug Administration today approved Exondys 51 (eteplirsen) injection, the first drug approved to treat patients with Duchenne muscular dystrophy (DMD). Exondys 51 is specifically indicated for patients who have a confirmed mutation of the dystrophin gene amenable to exon 51 skipping, which affects about 13 percent of the population with DMD.

from Food and Drug Administration--Press Releases http://ift.tt/2cTaI9X
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