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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.
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; Müller-Felber, Wolfgang; Vill, Katharina; Blaschek, Astrid; Smitka, Martin; van der Stam, Lieske; Weiss, Katja; Winter, Benedikt; Goldhahn, Klaus; Plecko, Barbara; Horber, Veronka; Bernert, Günther; Husain, Ralf A; Rauscher, Christian; Trollmann, Regina; Garbade, Sven F; Hahn, Andreas; von der Hagen, Maja; Kaindl, Angela M.
Affiliation
  • Weiß C; Department of Pediatric Neurology, Charité-Universitätsmedizin Berlin, Berlin, Germany; Center for Chronically Sick Children, Charité-Universitätsmedizin Berlin, Berlin, Germany.
  • Ziegler A; Division of Child Neurology and Metabolic Medicine, Center for Child and Adolescent Medicine, University Hospital Heidelberg, Heidelberg, Germany.
  • Becker LL; Department of Pediatric Neurology, Charité-Universitätsmedizin Berlin, Berlin, Germany; Center for Chronically Sick Children, Charité-Universitätsmedizin Berlin, Berlin, Germany; Institute of Cell Biology and Neurobiology, Charité-Universitätsmedizin Berlin, Berlin, Germany.
  • Johannsen J; Department of Pediatrics, University Medical Center Hamburg-Eppendorf, Hamburg, Germany.
  • Brennenstuhl H; Division of Child Neurology and Metabolic Medicine, Center for Child and Adolescent Medicine, University Hospital Heidelberg, Heidelberg, Germany.
  • Schreiber G; Department of Pediatric Neurology, Klinikum Kassel, Kassel, Germany.
  • Flotats-Bastardas M; Department of Pediatric Neurology, University Hospital Homburg, Homburg, Germany.
  • Stoltenburg C; Department of Pediatric Neurology, Charité-Universitätsmedizin Berlin, Berlin, Germany; Center for Chronically Sick Children, Charité-Universitätsmedizin Berlin, Berlin, Germany.
  • Hartmann H; Hannover Medical School, Clinic for Pediatric Kidney, Liver, and Metabolic Diseases, Hannover, Germany.
  • Illsinger S; Hannover Medical School, Clinic for Pediatric Kidney, Liver, and Metabolic Diseases, Hannover, Germany.
  • Denecke J; Department of Pediatrics, University Medical Center Hamburg-Eppendorf, Hamburg, Germany.
  • Pechmann A; Department of Neuropediatrics and Muscle Disorders, Medical Center, University of Freiburg, Freiburg, Germany.
  • Müller-Felber W; Department of Paediatric Neurology and Developmental Medicine, Hauner Children's Hospital, Ludwig Maximilian University of Munich, Munich, Germany.
  • Vill K; Department of Paediatric Neurology and Developmental Medicine, Hauner Children's Hospital, Ludwig Maximilian University of Munich, Munich, Germany.
  • Blaschek A; Department of Paediatric Neurology and Developmental Medicine, Hauner Children's Hospital, Ludwig Maximilian University of Munich, Munich, Germany.
  • Smitka M; Department of Neuropediatrics, Medical Faculty Carl Gustav Carus, Technical University Dresden, Dresden, Germany.
  • van der Stam L; Department of Pediatric Neurology, Charité-Universitätsmedizin Berlin, Berlin, Germany; Center for Chronically Sick Children, Charité-Universitätsmedizin Berlin, Berlin, Germany.
  • Weiss K; Department of Pediatric Cardiology, Charité-Universitätsmedizin Berlin, Berlin, Germany.
  • Winter B; Department of Pediatrics, Ulm University, Ulm, Germany.
  • Goldhahn K; Department of Pediatrics and Neuropediatrics, DRK Klinikum Westend, Berlin, Germany.
  • Plecko B; Department of Pediatrics and Adolescent Medicine, Division of General Pediatrics, Medical University Graz, Graz, Austria.
  • Horber V; Department of Paediatric Neurology, University Children's Hospital, Tübingen, Germany.
  • Bernert G; Department of Pediatrics, Klinik Favoriten, Vienna, Austria.
  • Husain RA; Department of Neuropediatrics, Jena University Hospital, Jena, Germany.
  • Rauscher C; Department of Pediatrics, Paracelsus Medical University, Salzburg, Austria.
  • Trollmann R; Department of Pediatrics, Division of Pediatric Neurology, Friedrich-Alexander University of Erlangen-Nürnberg, Erlangen, Germany.
  • Garbade SF; Division of Child Neurology and Metabolic Medicine, Center for Child and Adolescent Medicine, University Hospital Heidelberg, Heidelberg, Germany.
  • Hahn A; Department of Child Neurology, University Hospital, Gießen, Germany.
  • von der Hagen M; Department of Neuropediatrics, Medical Faculty Carl Gustav Carus, Technical University Dresden, Dresden, Germany.
  • Kaindl AM; Department of Pediatric Neurology, Charité-Universitätsmedizin Berlin, Berlin, Germany; Center for Chronically Sick Children, Charité-Universitätsmedizin Berlin, Berlin, Germany; Institute of Cell Biology and Neurobiology, Charité-Universitätsmedizin Berlin, Berlin, Germany. Electronic address: ange
Lancet Child Adolesc Health ; 6(1): 17-27, 2022 01.
Article in En | MEDLINE | ID: mdl-34756190
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Full text: 1 Collection: 01-internacional Database: MEDLINE Main subject: Oligonucleotides / Body Weight / Muscular Atrophy, Spinal / Genetic Therapy Type of study: Clinical_trials / Etiology_studies / Guideline / Observational_studies Limits: Child, preschool / Female / Humans / Infant / Male Country/Region as subject: Europa Language: En Journal: Lancet Child Adolesc Health Year: 2022 Document type: Article Affiliation country:
Fulltext
Add to My VHL
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Full text: 1 Collection: 01-internacional Database: MEDLINE Main subject: Oligonucleotides / Body Weight / Muscular Atrophy, Spinal / Genetic Therapy Type of study: Clinical_trials / Etiology_studies / Guideline / Observational_studies Limits: Child, preschool / Female / Humans / Infant / Male Country/Region as subject: Europa Language: En Journal: Lancet Child Adolesc Health Year: 2022 Document type: Article Affiliation country: