Efficacy fi cacy 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

Weiss, Claudia; Becker, Lena-Luise; Friese, Johannes; Blaschek, Astrid; Hahn, Andreas; Illsinger, Sabine; Schwartz, Oliver; Bernert, Guenther; von der Hagen, Maja; 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; Eisenkoelbl, Astrid; Koehler, Cornelia; Stettner, Georg M.; Cirak, Sebahattin; Hasselmann, Oswald; Kaindl, Angela M.; Garbade, Sven F.; Johannsen, Jessika; Ziegler, Andreas

Efficacy fi cacy 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

Author:

Weiss, Claudia; Becker, Lena-Luise; Friese, Johannes; Blaschek, Astrid; Hahn, Andreas; Illsinger, Sabine; Schwartz, Oliver; Bernert, Guenther; von der Hagen, Maja; 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; Eisenkoelbl, Astrid; Koehler, Cornelia; Stettner, Georg M.; Cirak, Sebahattin; Hasselmann, Oswald; Kaindl, Angela M.; Garbade, Sven F.; Johannsen, Jessika; Ziegler, Andreas

Tübinger Autor(en):

Horber, Veronka

Published in:

Lancet Regional Health - Europe (2024), Bd. 47, Article 101092

Verlagsangabe:

Amsterdam : Elsevier

Language:

English

Full text:

http://dx.doi.org/10.1016/j.lanepe.2024.101092

ISSN:

2666-7762