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A retrospective, non-interventional cohort study to compare adults with SMA receiving risdiplamwith untreated patients: Study design.
Publication Type: Scientific poster, presentation or proceeding Authors: Simpson, A.; Batech, M.; Carmona, A.; Lee, S.; Poll, A.; Edwards, S.; Guittari, C.; Sutherland, C.; Marini-Bettolo, C.; ...