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ICER Releases Draft Evidence Report on Treatments for Duchenne Muscular Dystrophy


The Institute for Clinical and Economic Review (ICER) today released a Draft Evidence Report assessing the clinical effectiveness and value of two gene therapies to treat Duchenne muscular dystrophy (DMD) — eteplirsen (Exondys 51®, Sarepta) and golodirsen (Sarepta) — as well as deflazacort (Emflaza®, PTC Therapeutics), a corticosteroid.

This draft report will be open to public comment until 5pm ET on June 18, 2019. Based on stakeholder feedback, ICER may revise key assumptions and findings for its Evidence Report, which will be published on July 11, 2019.  The Evidence Report will be subject to deliberation during a public meeting of the New England Comparative Effectiveness Public Advisory Council (New England CEPAC), one of ICER’s three independent evidence appraisal committees, on July 25, 2019.

ICER Releases Draft Evidence Report On Treatments For Duchenne Muscular Dystrophy