Editors' note: Evidence in focus: Nusinersen use in spinal muscular atrophy: Report of the Guideline Development, Dissemination, and Implementation Subcommittee of the American Academy of Neurology

In the article “Evidence in focus: Nusinersen use in spinal muscular atrophy,” Michelson et al. systematically reviewed 4 published clinical trials of nusinersen for patients with spinal muscular atrophy (SMA) and concluded that the evidence is highest for treatment of infantile- and childhood-onset SMA in the early and middle symptomatic phases.

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