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(Università Cattolica del Sacro Cuore), Katie Groves(Novartis (United States)), Andrea Seferian, Nuno Mendonça, Giovanni Baranello, Veronica Schembri, Odile Boespflug‐Tanguy(Inserm), Giorgia Coratti, Francesca Magri, Volker Straub(Newcastle upon Tyne Hospitals NHS Foundation Trust), Rita De Sanctis(Humanitas University), Mariacristina Scoto(University College London), Michaela Foà(Novartis (United Kingdom)), Giacomo P. Comi(University of Milan), Valentine Tahon, Simone Morando, S. Mouffak, Maria Carmela Pera(Università Cattolica del Sacro Cuore), Emanuela Pagliano(Fondazione IRCCS Istituto Neurologico Carlo Besta), Alessandra Govoni(Azienda Ospedaliero-Universitaria Careggi), Sitra Tauscher‐Wisniewski(Novartis (United States)), Arnaud Vanlander(Ghent University Hospital), Marika Pane(Università Cattolica del Sacro Cuore), Valeria Parente(University of Pavia), Deepa H. Chand(Akron Children's Hospital), Stefania Corti(University of Milan), Laurent Servais(University of Oxford), Marina Pedemonte(Istituto Giannina Gaslini), Anovick Jonas, A. Jollet, Olga Schneider, Aurore Daron(Centre Hospitalier Universitaire de Liège), Maria Teresa Arnoldi, Nicolas Deconinck(Université Libre de Bruxelles), Anna Mandelli, Haojun Ouyang(Novartis (United States)), Arseniy Lavrov(Novartis (United Kingdom)), Francesco Muntoni(Great Ormond Street Hospital), Riccardo Zanin, A.C. Defeldre(St. Louis Children's Hospital), Fabian Dal Farra(Newcastle upon Tyne Hospitals NHS Foundation Trust), Noemi Brolatti(Novartis (United Kingdom)), Laura Antonaci, Claudio Bruno(Istituto Giannina Gaslini), Shotaro Tachibana, S. De Lucia, Riccardo Masson, L. Buscemi(Novartis (United Kingdom)), Elke De Vos
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