View in EDS

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.

Saved in:
Bibliographic Details
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.
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
FullText Text:
  Availability: 0
Header DbId: mdl
DbLabel: MEDLINE Ultimate
An: 39434961
AccessLevel: 2
PubType: Academic Journal
PubTypeId: academicJournal
PreciseRelevancyScore: 0
IllustrationInfo
Items – Name: Title
  Label: Title
  Group: Ti
  Data: 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.
– Name: Author
  Label: Authors
  Group: Au
  Data: <searchLink fieldCode="AU" term="%22Weiß+C%22">Weiß C</searchLink>; 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.<br /><searchLink fieldCode="AU" term="%22Becker+LL%22">Becker LL</searchLink>; 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.<br /><searchLink fieldCode="AU" term="%22Friese+J%22">Friese J</searchLink>; 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.<br /><searchLink fieldCode="AU" term="%22Blaschek+A%22">Blaschek A</searchLink>; 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.<br /><searchLink fieldCode="AU" term="%22Hahn+A%22">Hahn A</searchLink>; Department of Child Neurology, University Hospital, Rudolf-Buchheim-Str. 8, Gießen 35392, Germany.<br /><searchLink fieldCode="AU" term="%22Illsinger+S%22">Illsinger S</searchLink>; Clinic for Pediatric Kidney-, Liver- and Metabolic Diseases, Hannover Medical School, Carl-Neuberg Str. 1, Hannover 30625, Germany.<br /><searchLink fieldCode="AU" term="%22Schwartz+O%22">Schwartz O</searchLink>; Department of Pediatric Neurology, University Hospital, Albert-Schweitzer-Strasse 33, Münster, Germany.<br /><searchLink fieldCode="AU" term="%22Bernert+G%22">Bernert G</searchLink>; Department of Pediatrics, Klinik Favoriten, Kundratstr. 3, Vienna 1100, Austria.<br /><searchLink fieldCode="AU" term="%22Hagen+MV%22">Hagen MV</searchLink>; Department of Neuropediatrics, Medizinische Fakultät Carl Gustav Carus, Technische Universität Dresden, Fetscherstr. 74, Dresden 01307, Germany.<br /><searchLink fieldCode="AU" term="%22Husain+RA%22">Husain RA</searchLink>; Department of Neuropediatrics, Jena University Hospital, Bachstr. 18, Jena 07743, Germany.<br /><searchLink fieldCode="AU" term="%22Goldhahn+K%22">Goldhahn K</searchLink>; Department of Pediatrics and Neuropediatrics, DRK Klinikum Westend, Spandauer Damm 130, Berlin 14050, Germany.<br /><searchLink fieldCode="AU" term="%22Kirschner+J%22">Kirschner J</searchLink>; Department of Neuropediatrics and Muscle Disorders, Medical Center - University of Freiburg, Faculty of Medicine, University of Freiburg, Heiliggeist-Str. 1, Freiburg 79106, Germany.<br /><searchLink fieldCode="AU" term="%22Pechmann+A%22">Pechmann A</searchLink>; Department of Neuropediatrics and Muscle Disorders, Medical Center - University of Freiburg, Faculty of Medicine, University of Freiburg, Heiliggeist-Str. 1, Freiburg 79106, Germany.<br /><searchLink fieldCode="AU" term="%22Flotats-Bastardas+M%22">Flotats-Bastardas M</searchLink>; University Hospital Homburg, Department of Pediatric Neurology, Kirrberger Str. 100, Homburg 66421, Germany.<br /><searchLink fieldCode="AU" term="%22Schreiber+G%22">Schreiber G</searchLink>; Klinikum Kassel, Department of Pediatric Neurology, Mönchebergstr. 41-43, Kassel 34125, Germany.<br /><searchLink fieldCode="AU" term="%22Schara+U%22">Schara U</searchLink>; Department of Pediatric Neurology, Centre for Neuromuscular Disorders, Center for Translational Neuro and Behavioral Sciences, University Duisburg-Essen, Germany.<br /><searchLink fieldCode="AU" term="%22Plecko+B%22">Plecko B</searchLink>; Department of Pediatrics and Adolescent Medicine, Division of General Pediatrics, Medical University Graz, Auenbruggerplatz 2, Graz 8036, Austria.<br /><searchLink fieldCode="AU" term="%22Trollmann+R%22">Trollmann R</searchLink>; Department of Pediatrics, Division of Pediatric Neurology, Friedrich-Alexander University of Erlangen-Nürnberg, Maximiliansplatz 2, Erlangen 91054, Germany.<br /><searchLink fieldCode="AU" term="%22Horber+V%22">Horber V</searchLink>; Department of Pediatric Neurology, University Children's Hospital, Hoppe-Seyler-Str. 1, Tübingen 72076, Germany.<br /><searchLink fieldCode="AU" term="%22Wilichowski+E%22">Wilichowski E</searchLink>; Department of Paediatrics and Adolescent Medicine, Division of Paediatric Neurology, University Medical Centre Göttingen, Georg August University Göttingen, Germany.<br /><searchLink fieldCode="AU" term="%22Baumann+M%22">Baumann M</searchLink>; Department of Pediatrics I, Division of Pediatric Neurology, Medical University Innsbruck, Innsbruck, Austria.<br /><searchLink fieldCode="AU" term="%22Klein+A%22">Klein A</searchLink>; Division of Neuropediatrics, Development and Rehabilitation, Department of Pediatrics, Inselspital, Bern University Hospital, University of Bern, Bern, Switzerland.<br /><searchLink fieldCode="AU" term="%22Eisenkölbl+A%22">Eisenkölbl A</searchLink>; Department of Paediatrics and Adolescent Medicine, Johannes Kepler University Linz, Kepler University Hospital, Krankenhausstrasse 26-30, Linz 4020, Austria.<br /><searchLink fieldCode="AU" term="%22Köhler+C%22">Köhler C</searchLink>; Bochum Department of Neuropediatrics, University Children's Hospital, Ruhr-University Bochum, Bochum, Germany.<br /><searchLink fieldCode="AU" term="%22Stettner+GM%22">Stettner GM</searchLink>; Neuromuscular Center Zurich and Department of Pediatric Neurology, University Children's Hospital Zurich, University of Zurich, Steinwiesstrasse 75, Zurich CH-8032, Switzerland.<br /><searchLink fieldCode="AU" term="%22Cirak+S%22">Cirak S</searchLink>; Ulm University, Department of Pediatrics, Albert-Einstein-Allee 23, Ulm 89081, Germany.<br /><searchLink fieldCode="AU" term="%22Hasselmann+O%22">Hasselmann O</searchLink>; Department of Neuropediatrics, Children's Hospital of Eastern Switzerland, St. Gallen, Switzerland.<br /><searchLink fieldCode="AU" term="%22Kaindl+AM%22">Kaindl AM</searchLink>; 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.<br /><searchLink fieldCode="AU" term="%22Garbade+SF%22">Garbade SF</searchLink>; 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.<br /><searchLink fieldCode="AU" term="%22Johannsen+J%22">Johannsen J</searchLink>; University Medical Center Hamburg-Eppendorf, Department of Pediatrics, Martinistr. 52, Hamburg 20246, Germany.<br /><searchLink fieldCode="AU" term="%22Ziegler+A%22">Ziegler A</searchLink>; 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.
– Name: AuthorCorporate
  Label: Corporate Authors
  Group: Au
  Data: <searchLink fieldCode="CA" term="%22SMArtCARE+and+Swiss-Reg-NMD+study+group%22">SMArtCARE and Swiss-Reg-NMD study group</searchLink>
– Name: TitleSource
  Label: Source
  Group: Src
  Data: <searchLink fieldCode="JN" term="%22101777707%22">The Lancet regional health. Europe</searchLink> [Lancet Reg Health Eur] 2024 Oct 07; Vol. 47, pp. 101092. <i>Date of Electronic Publication: </i>2024 Oct 07 (<i>Print Publication: </i>2024).
– Name: TypePub
  Label: Publication Type
  Group: TypPub
  Data: Journal Article
– Name: TitleSource
  Label: Journal Info
  Group: Src
  Data: <i>Publisher: </i><searchLink fieldCode="PB" term="%22[Oxford]%22">[Oxford] </searchLink><i>Country of Publication: </i>England <i>NLM ID: </i>101777707 <i>Publication Model: </i>eCollection <i>Cited Medium: </i>Internet <i>ISSN: </i>2666-7762 (Electronic) <i>Linking ISSN: </i><searchLink fieldCode="IS" term="%2226667762%22">26667762 </searchLink><i>NLM ISO Abbreviation: </i>Lancet Reg Health Eur <i>Subsets: </i>PubMed not MEDLINE
PLink https://search.ebscohost.com/login.aspx?direct=true&site=eds-live&db=mdl&AN=39434961
RecordInfo BibRecord:
  BibEntity:
    Identifiers:
      – Type: doi
        Value: 10.1016/j.lanepe.2024.101092
    Languages:
      – Code: eng
        Text: English
    PhysicalDescription:
      Pagination:
        StartPage: 101092
    Titles:
      – TitleFull: 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.
        Type: main
  BibRelationships:
    HasContributorRelationships:
      – PersonEntity:
          Name:
            NameFull: Weiß C
      – PersonEntity:
          Name:
            NameFull: Becker LL
      – PersonEntity:
          Name:
            NameFull: Friese J
      – PersonEntity:
          Name:
            NameFull: Blaschek A
      – PersonEntity:
          Name:
            NameFull: Hahn A
      – PersonEntity:
          Name:
            NameFull: Illsinger S
      – PersonEntity:
          Name:
            NameFull: Schwartz O
      – PersonEntity:
          Name:
            NameFull: Bernert G
      – PersonEntity:
          Name:
            NameFull: Hagen MV
      – PersonEntity:
          Name:
            NameFull: Husain RA
      – PersonEntity:
          Name:
            NameFull: Goldhahn K
      – PersonEntity:
          Name:
            NameFull: Kirschner J
      – PersonEntity:
          Name:
            NameFull: Pechmann A
      – PersonEntity:
          Name:
            NameFull: Flotats-Bastardas M
      – PersonEntity:
          Name:
            NameFull: Schreiber G
      – PersonEntity:
          Name:
            NameFull: Schara U
      – PersonEntity:
          Name:
            NameFull: Plecko B
      – PersonEntity:
          Name:
            NameFull: Trollmann R
      – PersonEntity:
          Name:
            NameFull: Horber V
      – PersonEntity:
          Name:
            NameFull: Wilichowski E
      – PersonEntity:
          Name:
            NameFull: Baumann M
      – PersonEntity:
          Name:
            NameFull: Klein A
      – PersonEntity:
          Name:
            NameFull: Eisenkölbl A
      – PersonEntity:
          Name:
            NameFull: Köhler C
      – PersonEntity:
          Name:
            NameFull: Stettner GM
      – PersonEntity:
          Name:
            NameFull: Cirak S
      – PersonEntity:
          Name:
            NameFull: Hasselmann O
      – PersonEntity:
          Name:
            NameFull: Kaindl AM
      – PersonEntity:
          Name:
            NameFull: Garbade SF
      – PersonEntity:
          Name:
            NameFull: Johannsen J
      – PersonEntity:
          Name:
            NameFull: Ziegler A
    IsPartOfRelationships:
      – BibEntity:
          Dates:
            – D: 07
              M: 10
              Text: 2024 Oct 07
              Type: published
              Y: 2024
          Identifiers:
            – Type: issn-electronic
              Value: 2666-7762
          Numbering:
            – Type: volume
              Value: 47
          Titles:
            – TitleFull: The Lancet regional health. Europe
              Type: main
ResultId 1