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FEBRUARY 8, 2016

Advocates Pushing FDA To Review Duchenne Drug

By Gina Shaw
 
Patient advocates for Duchenne muscular dystrophy (DMD) are calling for a grass-roots effort to convince the FDA not to lose its momentum on reviewing eteplirsen (Sarepta Therapeutics Inc.), an experimental exon-skipping drug for the disorder that was supposed to be the focus of a Jan. 22 advisory committee hearing that got derailed by winter storm Jonas.
 
Although no safety issues were identified in an FDA staff committee assessment of eteplirsen, released on Jan.