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
Authors
Ralf A. Husain (ORCID: https://orcid.org/0000-0003-0798-6346)
Stiftung für die Erforschung der Muskelkrankheiten
PTC Therapeutics
AveXis
Sarepta Therapeutics
Deutsche Gesellschaft für Muskelkranke
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article
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
Ralf A. Husain, Matthias Baumann, Marina Flotats‐Bastardas, Ekkehard Wilichowski et al.
Citations
22 citations
Journal
The Lancet Regional Health - Europe
Topics
Neurogenetic and Muscular Disorders Research
Field-Weighted Citation Impact (FWCI)
9.63
article
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
Ralf A. Husain, Matthias Baumann, Marina Flotats‐Bastardas, Ekkehard Wilichowski, Klaus Goldhahn, Janbernd Kirschner, Andrea Klein, Astrid Eisenkölbl, Veronka Horber, Regina Trollmann, Sebahattin Çırak, Andreas Ziegler, Georg Martin Stettner, Johannes Friese, Andreas Hahn, Gudrun Schreiber, Sabine Illsinger, Astrid Blaschek, Jessika Johannsen, Günther Bernert, Maja von der Hagen, Oswald Hasselmann, Lena‐Luise Becker, Barbara Plecko, Angela M. Kaindl, Sven F. Garbade, Cornelia Köhler, Claudia Weiß, Astrid Pechmann, Oliver Schwartz, Ulrike Schara
article en
22 citations
Abstract
No abstract available for this paper.
Publication Tag
The Lancet Regional Health - EuropeVol. 47
Institutions
University of Bern (CH), Johannes Kepler University of Linz (AT), Innsbruck Medical University (AT), Universität Hamburg (DE), University of Freiburg (DE), University of Mannheim (DE), Friedrich-Alexander-Universität Erlangen-Nürnberg (DE), Universität Ulm (DE), Medical University of Graz (AT), Heidelberg University (DE), University Hospital Bonn (DE), University Hospitals of the Ruhr-University of Bochum (DE), University Hospital of Bern (CH), University Medical Center Freiburg (DE), University Hospital Heidelberg (DE), Medizinische Hochschule Hannover (DE), Universitätsklinikum des Saarlandes (DE), University Hospital Münster (DE), University Medical Center Hamburg-Eppendorf (DE), Kepler Universitätsklinikum (AT), University Children's Hospital Tübingen (DE), University Children's Hospital Zurich (CH), DRK Kliniken Berlin (DE), Jena University Hospital (DE), Berlin Institute of Health at Charité - Universitätsmedizin Berlin (DE), Klinikum Kassel (DE), LWL-Universitätsklinikum Bochum (DE), University Hospital Carl Gustav Carus (DE), Technische Hochschule Ulm (DE), SBA Research (AT), University of Duisburg-Essen (DE), University of Göttingen (DE), Charité - Universitätsmedizin Berlin (DE), Ludwig-Maximilians-Universität München (DE)
Funders
Pfizer, Biogen, Stiftung für die Erforschung der Muskelkrankheiten, PTC Therapeutics, AveXis, Sarepta Therapeutics, Deutsche Gesellschaft für Muskelkranke