Randomized Trialhigh evidence
High-dose nusinersen for spinal muscular atrophy: a phase 3 randomized trial.
Nature medicine·March 1, 2026·PMID 41634391
Why it mattersDespite disease-modifying therapies, many SMA patients have residual deficits. This trial provides evidence that a higher nusinersen dose may offer greater motor benefit without compromising safety, informing dosing strategies for both treatment-naive and experienced patients.
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