Positive efficacy and safety results with nursinersen treatment at 6 and 14 months of follow-up in adult patients with spinal muscular atrophy (SMA) were recently reported in a study published in Neuromuscular Disorders. The researchers found increases in the baseline Medical Research Council (MRC) strength scale and the median revised Hammersmith scale (RHS) scores.

“We report a longer-term observation of nusinersen treatment effects and safety in a large cohort of adult patients,” the authors wrote. “Our study further supports the efficacy and safety of nusinersen treatment in adult patients with SMA2 and SMA3, with modest improvement in muscle strength, and stabilization of motor function over a relatively long period of observation.”

To date, the only clinical trials investigating the effectiveness of nursinersen treatment in patients with SMA have been conducted in pediatric patients. However, several cohort studies have demonstrated a positive effect of nursinersen on adults with SMA in terms of motor function.

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Here, the research team enrolled 37 adult patients ≥18 years of age with long-standing SMA type 2 or 3 between March 2018 and September 2020. They compared baseline neurological impairment, respiratory and motor function, and side effects, with measurements at 6, 14, and 26 months of nursinersen therapy.

The only side effect recorded was headache after lumbar puncture. The baseline RHS improved from baseline until 6 months, and the MRC scale increased from baseline until visits 6 and 14 months later. There was no change in respiratory function over the study period.

The study results contribute to the growing body of evidence demonstrating the effectiveness and safety of nursinersen therapy in adult patients with SMA type 2 and 3, along with modest improvements in key clinical measurements.


Fainmesser Y, Drory VE, Ben-Shushan S, et al. Longer-term follow-up of nusinersen efficacy and safety in adult patients with spinal muscular atrophy types 2 and 3. Neuromuscul Disord. Published online April 13, 2022. doi:10.1016/j.nmd.2022.04.003