Long-term real-world outcomes of nusinersen treatment in patients with spinal muscular atrophy types I-III. (PubMed, Brain Dev) - Oct 6, 2026 - "Nusinersen was associated with early motor improvement and long-term stabilization, a clinically meaningful outcome given the progressive nature of SMA."
Journal • Real-world evidence • CNS Disorders • Genetic Disorders • Movement Disorders • Muscular Atrophy • Rare Diseases • SMN2
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