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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

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source: The Lancet. Child & Adolescent Health

year: 2022

authors: 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


Given the novelty of gene replacement therapy with onasemnogene abeparvovec in spinal muscular atrophy, efficacy and safety data are limited, especially for children older than 24 months, those weighing more than 8·5 kg, and those who have received nusinersen. We aimed to provide real-world data on motor function and safety after gene replacement therapy in different patient subgroups.

organization: Department of Pediatric Neurology, Charité-Universitätsmedizin Berlin, Berlin, Germany; Center for Chronically Sick Children, Charité-Universitätsmedizin Berlin, Berlin, Germany.

DOI: 10.1016/S2352-4642(21)00287-X

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