Briefing Summary: US FDA Posts Advisory Committee Materials for Sarepta’s Eteplirsen for Duchenne Muscular Dystrophy – JAN 22, 2016 (PCNS)
The US FDA has posted details, including company and FDA Briefing Materials, for the Friday, January 22, 2016 Central Nervous System Drugs Advisory Committee (PCNS) meeting. The Committee will discuss eteplirsen (injection for intravenous infusion), by Sarepta Therapeutics, Inc. (Sarepta), for the treatment of Duchenne muscular dystrophy (DMD) in patients who have a confirmed mutation of the DMD gene that is amenable to exon 51 skipping. In briefing materials for the meeting, it appears the proposed US trade name for eteplirsen is exondys 51.
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