Efficacy and safety of gene therapy with onasemnogene abeparvovec in children with spinal muscular atrophy in the D-A-CH-region: a population-based observational study.
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| Title: | Efficacy and safety of gene therapy with onasemnogene abeparvovec in children with spinal muscular atrophy in the D-A-CH-region: a population-based observational study. |
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| Authors: | Weiß C; Charité-Universitätsmedizin Berlin, Department of Pediatric Neurology, Augustenburger Platz 1, Berlin 13353, Germany.; Charité-Universitätsmedizin Berlin, Center for Chronically Sick Children, Augustenburger Platz 1, Berlin 13353, Germany.; Department of Child Neurology, University Hospital Bonn, Venusberg-Campus 1, Bonn 53127, Germany., Becker LL; Charité-Universitätsmedizin Berlin, Department of Pediatric Neurology, Augustenburger Platz 1, Berlin 13353, Germany.; Charité-Universitätsmedizin Berlin, Center for Chronically Sick Children, Augustenburger Platz 1, Berlin 13353, Germany.; Charité-Universitätsmedizin Berlin, Institute of Cell Biology and Neurobiology, Augustenburger Platz 1, Berlin 13353, Germany.; Department of Child Neurology, University Hospital Bonn, Venusberg-Campus 1, Bonn 53127, Germany.; Department of Pediatric Neurology and Developmental Medicine, Ludwig Maximilian University of Munich (LMU), Hauner Children's Hospital, Lindwurmstr. 4, Munich 80337, Germany., Friese J; Charité-Universitätsmedizin Berlin, Center for Chronically Sick Children, Augustenburger Platz 1, Berlin 13353, Germany.; Department of Child Neurology, University Hospital Bonn, Venusberg-Campus 1, Bonn 53127, Germany., Blaschek A; Charité-Universitätsmedizin Berlin, Institute of Cell Biology and Neurobiology, Augustenburger Platz 1, Berlin 13353, Germany.; Department of Pediatric Neurology and Developmental Medicine, Ludwig Maximilian University of Munich (LMU), Hauner Children's Hospital, Lindwurmstr. 4, Munich 80337, Germany., Hahn A; Department of Child Neurology, University Hospital, Rudolf-Buchheim-Str. 8, Gießen 35392, Germany., Illsinger S; Clinic for Pediatric Kidney-, Liver- and Metabolic Diseases, Hannover Medical School, Carl-Neuberg Str. 1, Hannover 30625, Germany., Schwartz O; Department of Pediatric Neurology, University Hospital, Albert-Schweitzer-Strasse 33, Münster, Germany., Bernert G; Department of Pediatrics, Klinik Favoriten, Kundratstr. 3, Vienna 1100, Austria., Hagen MV; Department of Neuropediatrics, Medizinische Fakultät Carl Gustav Carus, Technische Universität Dresden, Fetscherstr. 74, Dresden 01307, Germany., Husain RA; Department of Neuropediatrics, Jena University Hospital, Bachstr. 18, Jena 07743, Germany., Goldhahn K; Department of Pediatrics and Neuropediatrics, DRK Klinikum Westend, Spandauer Damm 130, Berlin 14050, Germany., Kirschner J; Department of Neuropediatrics and Muscle Disorders, Medical Center - University of Freiburg, Faculty of Medicine, University of Freiburg, Heiliggeist-Str. 1, Freiburg 79106, Germany., Pechmann A; Department of Neuropediatrics and Muscle Disorders, Medical Center - University of Freiburg, Faculty of Medicine, University of Freiburg, Heiliggeist-Str. 1, Freiburg 79106, Germany., Flotats-Bastardas M; University Hospital Homburg, Department of Pediatric Neurology, Kirrberger Str. 100, Homburg 66421, Germany., Schreiber G; Klinikum Kassel, Department of Pediatric Neurology, Mönchebergstr. 41-43, Kassel 34125, Germany., Schara U; Department of Pediatric Neurology, Centre for Neuromuscular Disorders, Center for Translational Neuro and Behavioral Sciences, University Duisburg-Essen, Germany., Plecko B; Department of Pediatrics and Adolescent Medicine, Division of General Pediatrics, Medical University Graz, Auenbruggerplatz 2, Graz 8036, Austria., Trollmann R; Department of Pediatrics, Division of Pediatric Neurology, Friedrich-Alexander University of Erlangen-Nürnberg, Maximiliansplatz 2, Erlangen 91054, Germany., Horber V; Department of Pediatric Neurology, University Children's Hospital, Hoppe-Seyler-Str. 1, Tübingen 72076, Germany., Wilichowski E; Department of Paediatrics and Adolescent Medicine, Division of Paediatric Neurology, University Medical Centre Göttingen, Georg August University Göttingen, Germany., Baumann M; Department of Pediatrics I, Division of Pediatric Neurology, Medical University Innsbruck, Innsbruck, Austria., Klein A; Division of Neuropediatrics, Development and Rehabilitation, Department of Pediatrics, Inselspital, Bern University Hospital, University of Bern, Bern, Switzerland., Eisenkölbl A; Department of Paediatrics and Adolescent Medicine, Johannes Kepler University Linz, Kepler University Hospital, Krankenhausstrasse 26-30, Linz 4020, Austria., Köhler C; Bochum Department of Neuropediatrics, University Children's Hospital, Ruhr-University Bochum, Bochum, Germany., Stettner GM; Neuromuscular Center Zurich and Department of Pediatric Neurology, University Children's Hospital Zurich, University of Zurich, Steinwiesstrasse 75, Zurich CH-8032, Switzerland., Cirak S; Ulm University, Department of Pediatrics, Albert-Einstein-Allee 23, Ulm 89081, Germany., Hasselmann O; Department of Neuropediatrics, Children's Hospital of Eastern Switzerland, St. Gallen, Switzerland., Kaindl AM; Charité-Universitätsmedizin Berlin, Department of Pediatric Neurology, Augustenburger Platz 1, Berlin 13353, Germany.; Charité-Universitätsmedizin Berlin, Center for Chronically Sick Children, Augustenburger Platz 1, Berlin 13353, Germany.; Charité-Universitätsmedizin Berlin, Institute of Cell Biology and Neurobiology, Augustenburger Platz 1, Berlin 13353, Germany.; Department of Child Neurology, University Hospital Bonn, Venusberg-Campus 1, Bonn 53127, Germany.; Department of Pediatric Neurology and Developmental Medicine, Ludwig Maximilian University of Munich (LMU), Hauner Children's Hospital, Lindwurmstr. 4, Munich 80337, Germany., Garbade SF; Heidelberg University, Medical Faculty Heidelberg, Center for Pediatric and Adolescent Medicine, Department I, Division of Pediatric Neurology and Metabolic Medicine, Im Neuenheimer Feld 430, Heidelberg 69120, Germany., Johannsen J; University Medical Center Hamburg-Eppendorf, Department of Pediatrics, Martinistr. 52, Hamburg 20246, Germany., Ziegler A; Heidelberg University, Medical Faculty Heidelberg, Center for Pediatric and Adolescent Medicine, Department I, Division of Pediatric Neurology and Metabolic Medicine, Im Neuenheimer Feld 430, Heidelberg 69120, Germany. |
| Corporate Authors: | SMArtCARE and Swiss-Reg-NMD study group |
| Source: | The Lancet regional health. Europe [Lancet Reg Health Eur] 2024 Oct 07; Vol. 47, pp. 101092. Date of Electronic Publication: 2024 Oct 07 (Print Publication: 2024). |
| Publication Type: | Journal Article |
| Journal Info: | Publisher: [Oxford] Country of Publication: England NLM ID: 101777707 Publication Model: eCollection Cited Medium: Internet ISSN: 2666-7762 (Electronic) Linking ISSN: 26667762 NLM ISO Abbreviation: Lancet Reg Health Eur Subsets: PubMed not MEDLINE |
| Database: | MEDLINE Ultimate |
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