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ICER Document Reviews Effectiveness of 3 Duchenne MD Therapies


The Institute for Clinical and Economic Review (ICER) has set up a draft document to review evidence of the effectiveness of treatments for Duchenne muscular dystrophy (DMD).

The review, “Deflazacort, Eteplirsen, and Golodirsen for Duchenne Muscular Dystrophy: Effectiveness and Value,” focuses on Sarepta Therapeutics’ Exondys 51 (eteplirsen) and Golodirsen (SRP-4053), and on PTC Therapeutics’ Emflaza (deflazacort), and will include input from diverse stakeholders, including patients and their families, researchers, and the therapies’ developers.

ICER Document Reviews Effectiveness Of 3 Duchenne MD Therapies