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1. S12 Onasemnogene abeparvovec gene therapy for spinal muscular atrophy type 1: phase 3 study (STR1VE-US)

Author

Day, JW, primary, Finkel, RS, additional, Connolly, AM, additional, Darras, BT, additional, Iannaccone, ST, additional, Kuntz, NL, additional, Peña, LDM, additional, Smith, EC, additional, Chiriboga, CA, additional, Crawford, TO, additional, Shieh, PB, additional, Kwon, JM, additional, Zaidman, CM, additional, Schultz, M, additional, Kausar, I, additional, Chand, D, additional, Tauscher-Wisniewski, S, additional, Ouyang, H, additional, Macek, TA, additional, and Mendell, JR, additional

Published
2021
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