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

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

Publisher: Elsevier BV

Year: 2021

Pages: 10

DOI: 10.1016/s1474-4422(21)00251-9

DOI owner: Elsevier


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