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2025-09-10
13:03

[DKFZ-2025-01871] Journal Article

Wurster, C. D. ; Stolte, B. ; Kessler, T. ; et al
Respiratory function in 192 adult patients with spinal muscular atrophy (SMA) treated with nusinersen - a multicenter observational study.

Orphanet journal of rare diseases 20(1), 476 (2025) [10.1186/s13023-025-04009-3]

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. [...]

ddc:610

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