Topic: Spinal Muscular Atrophy

Spinal Muscular Atrophy

Mar 2019 | Assessment

Treatments of Interest: onasemnogene abeparvovec (Zolgensma®, Novartis AG/AveXis) nusinersen (Spinraza®, Biogen) Spinal muscular atrophy (SMA) is a rare, genetic neuromuscular disease. In its most common form, Type I SMA (~60% of cases), symptoms begin in infancy. Affected children never sit up, and they rapidly develop difficulty swallowing and breathing, eventually requiring feeding tubes and mechanical […]