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Intravenous and Intrathecal Onasemnogene Abeparvovec Gene Therapy in Symptomatic and Presymptomatic Spinal Muscular Atrophy: 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. LIPPINCOTT WILLIAMS & WILKINS. 2023
View details for DOI 10.1212/WNL.0000000000202555
View details for Web of Science ID 001053672107192