Reader Response: Nusinersen in Adult Patients With Spinal Muscular Atrophy: Observations From a Single Center

Moshe-Lilie et al.1 retrospectively reviewed the charts of 22 patients with spinal muscular atrophy (SMA) 2 and SMA 3, 10 of whom received nusinersen. Nine patients had SMA 2, and 13 had SMA 3. Most patients had a long-standing disease duration, were wheelchair-bound, and had severe scoliosis, and half of the patients had respiratory problems. Patients were assessed mainly with Medical Research Council (MRC) scoring, and the median follow-up was 1 year. End points were objective motor gain or stability. The results of the study did not show objective improvement. In a relevant number of these few patients, lumbar puncture-associated severe side effects occurred. The authors conclude that the risk-benefit ratio of nusinersen in adult patients needs critical decision making.

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