Onasemnogene abeparvovec gene therapy for symptomatic infantile-onset spinal muscular atrophy type 1 (STR1VE-EU): an open-label, single-arm, multicentre, phase 3 trial

Eugenio Maria Mercuri, Francesco Muntoni, Giovanni Baranello, Riccardo Masson, Odile Boespflug-Tanguy, Claudio Bruno, Stefania Corti, Aurore Daron, Nicolas Deconinck, Laurent Servais, Volker Straub, Haojun Ouyang, Deepa Chand, Sitra Tauscher-Wisniewski, Nuno Mendonca, Arseniy Lavrov, A. Seferian, S. De Lucia, S. Tachibana, A. JolletS. Mouffak, M. Pedemonte, N. Brolatti, S. Morando, A. Vanlander, E. De Vos, V. Tahon, A. Govoni, F. Magri, G. Comi, M. Foa, V. Parente, L. Buscemi, F. Dal Farra, O. Schneider, A. Jonas, A. C. Defeldre, E. Pagliano, R. Zanin, M. T. Arnoldi, V. Schembri, M. Del Sole, A. Mandelli, Maria Carmela Pera, L. Antonaci, Laura Antonaci, Giorgia Coratti, R. De Sanctis, Roberto De Sanctis, Marika Pane, M. Scoto, K. Groves, L. Edel, F. Abel, H. Van Ruiten, R. M. Lofra, E. Thompson

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