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Machine learning predicts treatment response to nusinersen in non-sitter Spinal Muscular Atrophy (SMA).

2025-10-23 neurology Title + abstract only
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1.1BackgroundNusinersen has substantially increased survival and improved disease progression in Spinal Muscular Atrophy (SMA) patients. However, treatment response is heterogeneous, with some patients gaining the ability to sit and walk whilst others display less obvious changes. MethodFrom the SMA Reach UK and the Italian Telethon Network, 124 non-sitter SMA patients treated with nusinersen under 4 years were included and randomly allocated to training and testing (80/20%). Tree and regressio...

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