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
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