Duchenne Muscular Dystrophy
ICER will assess the comparative clinical effectiveness and value of treatments for Duchenne muscular dystrophy. This evidence review will examine deflazacort (Emflaza®, PTC Therapeutics) and two exon-skipping therapies: eteplirsen (Exondys 51®, Sarepta) and golodirsen (Sarepta). Eteplirsen was approved by the FDA in September 2016 for patients with a confirmed mutation in the dystrophin gene that is amenable to exon 51 skipping. Sarepta has announced its intention to file for accelerated approval of golodirsen by the end of 2018.
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MIT Samberg Conference Center
50 Memorial Drive
Cambridge, MA 02142
The New England CEPAC will convene to deliberate and vote on evidence presented in ICER's report on treatments for Duchenne muscular dystrophy.