Key result
Disease-modifying therapies fail to eliminate scoliosis development or progression in spinal muscular atrophy.
Why the study?
Disease-modifying therapies have dramatically shifted the natural history of spinal muscular atrophy but do not eliminate the development or progression of scoliosis.
This review summarizes current evidence and clinical strategies for managing scoliosis in SMA patients, noting that while disease-modifying therapies improve survival and function, they do not eliminate scoliosis progression.
Spinal deformities persist in SMA despite disease-modifying therapies; leaves open optimal orthopedic strategies for non-ambulatory patients.
Spinal muscular atrophy (SMA) is a hereditary neuromuscular disorder characterized by degeneration of anterior horn cells in the spinal cord, resulting in progressive muscular weakness. Scoliosis is frequently encountered in SMA patients (especially in non-ambulatory patients), particularly in Types II and III. It contributes significantly to truncal instability, pulmonary complications, and reduced quality of life. With the advent of disease-modifying therapies such as Nusinersen, Risdiplam, and Onasemnogene abeparvovec, the natural history of SMA has shifted dramatically, leading to prolonged survival and functional improvement. However, these therapies do not eliminate the development or progression of scoliosis. This review aims to summarize the current evidence and clinical strategies for the management of scoliosis in SMA, including conservative interventions, surgical options, and the emerging influence of pharmacologic therapies.
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Basu et al. (2026) conducted a review in Spinal muscular atrophy (SMA) and scoliosis. Management of scoliosis (conservative, surgical, and pharmacologic) was evaluated. This review summarizes current evidence and clinical strategies for managing scoliosis in spinal muscular atrophy, noting that disease-modifying therapies do not eliminate its development or progression.
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