Emerging Literature on Multidisciplinary Care in Spinal Muscular Atrophy

Spinal muscular atrophy (SMA) is a rare autosomal recessive neuromuscular disorder caused by biallelic variants in the survival motor neuron 1 (SMN1) gene, leading to degeneration of motor neurons in the spinal cord and progressive proximal muscle weakness. The approval of 3 SMN-enhancing therapies, including nusinersen (Spinraza; Biogen), onasemnogene abeparvovec (Zolgensma; Novartis), and risdiplam (Evrysdi; Roche), has substantially altered the natural history of the disease, but it has not reduced the need for coordinated care across disciplines.