Machine learning predicts treatment response to nusinersen in non-sitter spinal muscular atrophy (SMA). (PubMed, J Neuromuscul Dis) - Oct 21, 2026 - "Specific motor function features, including the ability to kick in supine, were strongly associated with a higher likelihood of sitting.InterpretationThis work provides a framework for predicting nusinersen-response and represents the first stage in personalised counselling on treatment plans for SMA.FundingSupport for SMA Reach is provided by Biogen, Roche and Novartis, and historically the NIHR BRC, SMA Trust, the MRC Translational Research Centre and MD UK. Support for the Italian network is provided by Famiglie SMA, Telethon (GSP 13002), and ASAMSI."
Journal • Genetic Disorders • Movement Disorders • Muscular Atrophy • Rare Diseases
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; Deborah Ridout; Emer O'Reilly; Eugenio Mercuri; Francesco Muntoni; Georgia Stimpson; Giorgia Coratti; Giovanni Baranello; Mariacristina Scoto; Marika Pane; Roberto de Sanctis; Robin Mitra; Tapabrata Chakraborti
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