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

Abstract

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. In response, Takanashi et al. note that although no life-threatening adverse events were reported in the trials, 5 patients were noted to have communicating hydrocephalus. They present a 6-month-old boy with SMA type 1 who developed communicating hydrocephalus in temporal association with nusinersen treatment that subsequently improved with ventriculoperitoneal shunting. They conclude that communicating hydrocephalus is a rare but serious adverse event in nusinersen treatment. However, the authors of the article did not respond to this comment. 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.