Evaluation consultation document: Onasemnogene abeparvovec for treating spinal muscular atrophy

In DRAFT guidance, NICE is recommending onasemnogene abeparvovec (Zolgensma) as an option for treating 5q spinal muscular atrophy (SMA) in babies with type 1 SMA and for babies with SMA before they develop symptoms as part of a managed access arrangement.

Source:

National Institute for Health and Care Excellence