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3. 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 C; Ziegler A; Becker LL; Johannsen J; Brennenstuhl H; Schreiber G; Flotats-Bastardas M; Stoltenburg C; Hartmann H; Illsinger S; Denecke J; Pechmann A; Müller-Felber W; Vill K; Blaschek A; Smitka M; van der Stam L; Weiss K; Winter B; Goldhahn K; Plecko B; Horber V; Bernert G; Husain RA; Rauscher C; Trollmann R; Garbade SF; Hahn A; von der Hagen M; Kaindl AM Lancet Child Adolesc Health; 2022 Jan; 6(1):17-27. PubMed ID: 34756190 [TBL] [Abstract][Full Text] [Related]
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