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Onasemnogene abeparvovec gene therapy for symptomatic infantile-onset spinal muscular atrophy type 1 (STR1VE-EU): an open-label, single-arm, multicentre, phase 3 trial
Lancet Neurol 2021 Oct 01;20(10)832-841, E Mercuri, F Muntoni, G Baranello, R Masson, O Boespflug-Tanguy, C Bruno, S Corti, A Daron, N Deconinck, L Servais, V Straub, H Ouyang, D Chand, S Tauscher-Wisniewski, N Mendonca, A Lavrov