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