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Intravenous and intrathecal onasemnogene abeparvovec gene therapy in symptomatic and presymptomatic spinal muscular atrophy (SMA): long-term follow-up study Darras, B., Mercuri, E., Strauss, K., Day, J., Chien, Y., Masson, R., Wigderson, M., Alecu, I., Ballarini, N., Mehl, L., Marra, J., Connolly, A. PERGAMON-ELSEVIER SCIENCE LTD. 2023: S87
View details for DOI 10.1016/j.nmd.2023.07.093
View details for Web of Science ID 001087070800083