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New England CEPAC Meeting: Spinal Muscular Atrophy

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Omni Parker House

60 School Street

Boston, Massachusetts 02108

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The New England Comparative Effectiveness Public Advisory Council (New England CEPAC), a core program of the Institute for Clinical and Economic Review (ICER), will hold a public meeting on March 7, 2019 to consider the comparative clinical effectiveness and value of onasemnogene abeparvovec (Zolgensma®, Novartis AG/AveXis) and nusinersen (Spinraza®, Biogen) for treatment of spinal muscular atrophy (SMA).

SMA is a rare, genetic neuromuscular disease with the most severe cases affecting infants and young children. SMA incidence is approximately 1 in 10,000 live births or about 500 new SMA cases per year. The disease causes the irreversible degeneration of motor neurons, which leads to progressive muscle weakness and prevents patients from reaching motor milestones or retaining motor functions.

A list of key dates for stakeholder engagement opportunities and other important information associated with the review of onasemnogene abeparvovec and nusinersen for SMA can be found on ICER's website at https://icer-review.org/topic/sma/.

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Omni Parker House

60 School Street

Boston, Massachusetts 02108

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