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