Gene replacement therapy with onasemnogene abeparvovec in children with spinal muscular atrophy aged 24 months or younger and bodyweight up to 15 kg: an observational cohort study

Claudia Weiß, Andreas Ziegler, Lena-Luise Becker, Jessika Johannsen, Heiko Brennenstuhl, Gudrun Schreiber, Marina Flotats-Bastardas, Corinna Stoltenburg, Hans Hartmann, Sabine Illsinger, Jonas Denecke, Astrid Pechmann, Wolfgang Müller-Felber, Katharina Vill, Astrid Blaschek, Martin Smitka, Lieske van der Stam, Katja Weiss, Benedikt Winter, Klaus Goldhahn, Barbara Plecko, Veronka Horber, Günther Bernert, Ralf A Husain, Christian Rauscher, Regina Trollmann, Sven F Garbade, Andreas Hahn, Maja von der Hagen, Angela M Kaindl