Efficacy and safety of gene therapy with onasemnogene abeparvovec in children with spinal muscular atrophy in the D-A-CH-region: a population-based observational study
Titel:
Efficacy and safety of gene therapy with onasemnogene abeparvovec in children with spinal muscular atrophy in the D-A-CH-region: a population-based observational study
Auteur:
Weiß, Claudia Becker, Lena-Luise Friese, Johannes Blaschek, Astrid Hahn, Andreas Illsinger, Sabine Schwartz, Oliver Bernert, Günther Hagen, Maja von der Husain, Ralf A. Goldhahn, Klaus Kirschner, Janbernd Pechmann, Astrid Flotats-Bastardas, Marina Schreiber, Gudrun Schara, Ulrike Plecko, Barbara Trollmann, Regina Horber, Veronka Wilichowski, Ekkehard Baumann, Matthias Klein, Andrea Eisenkölbl, Astrid Köhler, Cornelia Stettner, Georg M. Cirak, Sebahattin Hasselmann, Oswald Kaindl, Angela M. Garbade, Sven F. Johannsen, Jessika Ziegler, Andreas