Transforming Spinal Muscular Atrophy: From Pivotal Trials to Real-World Evidence and Future Therapeutic Frontiers in Types 1 and 2.

Biomedicines · 2025 · PMC12383623 · PMID 40868194

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a positive trendno p-value reported

After nusinersen treatment, sitters—including all Type 2 patients—showed a mean HFMSE increase of 1.12 points at day 63, 2.32 points at day 180, and 3.21 points at day 300, with the change at day 63 reaching statistical significance ( p = 0.040) and a positive trend at subsequent time points.

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did not reach statistical significanceno p-value reported

Only CHOP-INTEND scores showed clinically meaningful change (increase in score by ≥4), but still did not reach statistical significance.

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an increasing trendno p-value reported

Significant improvements in motor function scores among those with Type 1 SMA ( p < 0.002) and Type 2 SMA ( p < 0.002) were reported; there was an increasing trend among those with Type 3 SMA, but it was not significant ( p = 0.051).

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