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- [1] A severely affected adult type 2 spinal muscular atrophy patient treated with risdiplam Neurological Sciences, 2023, 44 : 1449 - 1450
- [2] Risdiplam in Type 1 Spinal Muscular Atrophy NEW ENGLAND JOURNAL OF MEDICINE, 2021, 384 (10): : 915 - 923
- [9] Risdiplam-Treated Infants with Type 1 Spinal Muscular Atrophy versus Historical Controls NEW ENGLAND JOURNAL OF MEDICINE, 2021, 385 (05): : 427 - 435