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New England CEPAC Meeting: Duchenne Muscular Dystrophy

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MIT Samberg Conference Center

Salon I/T

50 Memorial Drive

Cambridge, MA 02142

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The New England Comparative Effectiveness Public Advisory Council (CEPAC), a core program of the Institute for Clinical and Economic Review (ICER), will hold a public meeting on July 25, 2019 to consider the comparative clinical effectiveness and value of deflazacort (Emflaza®, PTC Therapeutics), eteplirsen (Exondys 51®, Sarepta) and golodirsen (Sarepta) for the treatment of Duchenne muscular dystrophy (DMD).

DMD is a fatal, X-linked neuromuscular disease caused by mutations in the dystrophin gene that result in progressive loss of muscle function, including skeletal and cardiac function. It is the most common pediatric muscular dystrophy, affecting about 400 to 600 boys per year in the US.

This is ICER's first review of treatments for DMD. A presentation of the evidence will be followed by a public comment period and deliberations and votes by the New England CEPAC. The votes will be followed by a policy roundtable discussion.

A list of key dates for stakeholder engagement opportunities and other important information associated with the review of deflazacort, eteplirsen, and golodirsen for DMD can be found on ICER's website at https://icer-review.org/topic/duchenne-muscular-dystrophy/.

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Location

MIT Samberg Conference Center

Salon I/T

50 Memorial Drive

Cambridge, MA 02142

View Map

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