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Longitudinal Assessment of 4-Year HFMSE Changes in SMA II and III Patients Treated With Nusinersen.

Longitudinal Assessment of 4-Year HFMSE Changes in SMA II and III Patients Treated With Nusinersen. European journal of neurology Coratti, G., Bovis, F., Pane, M., Pasternak, A., Albamonte, E., Mizzoni, I., Glanzman, A. M., Morando, S., Montes, J., Cavallina, I., Dunaway Young, S., Duong, T., Rolle, E., Civitello, M., De Sanctis, R., Bravetti, C., Ricci, F., Gadaleta, G., Mongini, T., Sframeli, M., Pera, M. C., Messina, S., D'Amico, A., Catteruccia, M., Brolatti, N., Hirano, M., Zolkipli-Cunningham, Z., Darras, B. T., Bertini, E., Bruno, C., Day, J., Sansone, V. A., Finkel, R. S., Mercuri, E. 2025; 32 (7): e70268

Abstract

The aim of this international retrospective study was to assess 4-year change using the Hammersmith Functional Motor Scale Expanded (HFMSE) in individuals with type II and III spinal muscular atrophy (SMA) treated with nusinersen and to establish predictors of HFMSE changes.Individuals with type II or III SMA, and at least 4?years of nusinersen-only treatment were included. All were assessed using the HFMSE. Age at baseline, sex, motor function, SMN2 copy number, and age of onset were also retrospectively collected. Linear mixed effect models were used to calculate yearly changes and trajectory predictors.We included 73 individuals with SMA type II (mean age 8.58?years, SD 7.91, IQR 3.04-10.70) and 111 type III (mean age 7.91?years, SD 17.83, IQR 8.15-34.42). Over 4?years, mean changes were?+?4.18 (95% CI: 2.85-5.50) for SMA II and?+?1.08 (95% CI: 0.12-2.04) for SMA III. Age (SMA II: -0.34\[-0.51 to -0.17]; SMA III: -0.13\[-0.20 to -0.06], p?

View details for DOI 10.1111/ene.70268

View details for PubMedID 40576143