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PDF Availablehttps://doi.org/10.1136/archdischild-2023-325762
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Cite Journal: Archives of Disease in Childhood |  Publication Date: Jul 7, 2023 |
 Citations: 4 |
Affiliation: University of Toronto, Hospital for Sick Children
ObjectiveSpinal muscular atrophy (SMA) is a neuromuscular disorder that manifests with motor deterioration and respiratory complications. The paradigm of care is shifting as disease-modifying therapies including nusinersen, onasemnogene abeparvovec and...
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