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Onasemnogene Abeparvovec Gene Therapy for Infantile-Onset Spinal Muscular Atrophy in Patients With Two Copies of SMN2
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The Lancet Neurology
From MEDLINE®/PubMed®, a database of the U.S. National Library of Medicine.
Onasemnogene Abeparvovec Gene Therapy for Symptomatic Infantile-Onset Spinal Muscular Atrophy in Patients With Two Copies of SMN2 (STR1VE): An Open-Label, Single-Arm, Multicentre, Phase 3 Trial
Lancet Neurol 2021 Apr 01;20(4)284-293, JW Day, RS Finkel, CA Chiriboga, AM Connolly, TO Crawford, BT Darras, ST Iannaccone, NL Kuntz, LDM Peña, PB Shieh, EC Smith, JM Kwon, CM Zaidman, M Schultz, DE Feltner, S Tauscher-Wisniewski, H Ouyang, DH Chand, DM Sproule, TA Macek, JR MendellFrom MEDLINE®/PubMed®, a database of the U.S. National Library of Medicine.