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 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 H. Chand, Sitra Tauscher‐Wisniewski, Nuno Mendonça, Arseniy Lavrov, Andrea Seferian, S. De Lucia, Shotaro Tachibana, A. Jollet, S. Mouffak, Marina Pedemonte, Noemi Brolatti, Simone Morando, Arnaud Vanlander, Elke De Vos, Valentine Tahon, Alessandra Govoni, Francesca Magri, Giacomo P. Comi, Michaela Foà, Valeria Parente, L. Buscemi, Fabian Dal Farra, Olga Schneider, Anovick Jonas, A.C. Defeldre, Emanuela Pagliano, Riccardo Zanin, Maria Teresa Arnoldi, Veronica Schembri, M. Del Sole, Anna Mandelli, Maria Carmela Pera, Laura Antonaci, Giorgia Coratti, Rita De Sanctis, Marika Pane, Mariacristina Scoto, Katie Groves, Lisa Edel, François Abel, H. van Ruiten, R. Muni Lofra, E. T. Thompson