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Respiratory function in 192 adult patients with spinal muscular atrophy (SMA) treated with nusinersen : a multicenter observational…

Background: Natural history data show that respiratory function is impaired in SMA patients. Observational studies have shown stabilization of respiratory function in adult SMA patients treated with nusinersen. However, long-term studies investigating the effect of nusinersen on respiratory function…
London: BioMed Central, 2025-09-08