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A retrospective, non-interventional cohort study to compare adults with SMA receiving risdiplamwith untreated patients: Study design.
Walter, M.; Marlène Jagut; Haberlová, J.; Hodgkinson, V.; Yiu, E.; Murphy, L.; TREAT-NMD Global Data systems Oversight Committee (TGDOC); TREAT-NMD Global Registry Network for SMA; Roe, T.; ...