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

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; Muller-Felber, Wolfgang; Vill, Katharina; Blaschek, Astrid; Smitka, Martin; van der Stam, Lieske; Weiss, Katja; Winter, Benedikt; Goldhahn, Klaus; Plecko, Barbara; Horber, Veronka; Bernert, Gunther; Husain, Ralf A.; Rauscher, Christian; Trollmann, Regina; Garbade, Sven F.; Hahn, Andreas; von der Hagen, Maja; Kaindl, Angela M.

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

Autor(en):

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; Muller-Felber, Wolfgang; Vill, Katharina; Blaschek, Astrid; Smitka, Martin; van der Stam, Lieske; Weiss, Katja; Winter, Benedikt; Goldhahn, Klaus; Plecko, Barbara; Horber, Veronka; Bernert, Gunther; Husain, Ralf A.; Rauscher, Christian; Trollmann, Regina; Garbade, Sven F.; Hahn, Andreas; von der Hagen, Maja; Kaindl, Angela M.

Tübinger Autor(en):

Horber, Veronka

Erschienen in:

Lancet Child & Adolescent Health (2022), Bd. 6, H. 1, S. 17-27

Verlagsangabe:

Elsevier Sci Ltd

Sprache:

Englisch

Referenz zum Volltext:

http://dx.doi.org/10.1016/S2352-4642(21)00287-X

ISSN:

2352-4642