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Nusinersen is approved for treatment of all patients with spinal muscular atrophy (SMA), but its efficacy has been demonstrated in only infants and children. To address nusinersen efficacy in adults, investigators conducted an observational cohort study of 124 patients aged 16 to 65 years with SMA (mostly SMA type 2 or 3) treated with nusinersen, assessing them at 6, 10, and 14 months. Roughly one third of the patients were more mildly affected (able to ambulate). The primary outcome measure was change from baseline in the Hammersmith Functional Motor Scale Expanded (HFMSE) score, which consists of 33 motor functions that pertain to activities of daily living and has been used frequently in other SMA clinical trials.
The cohort had statistic…