Handlungsempfehlungen zur Gentherapie der spinalen Muskelatrophie mit Onasemnogene Abeparvovec – AVXS-101 View Full Text


Ontology type: schema:ScholarlyArticle      Open Access: True


Article Info

DATE

2020-05-11

AUTHORS

Andreas Ziegler, Ekkehard Wilichowski, Ulrike Schara, Andreas Hahn, Wolfgang Müller-Felber, Jessika Johannsen, Maja von der Hagen, Arpad von Moers, Corinna Stoltenburg, Afshin Saffari, Maggie C. Walter, Ralf A. Husain, Astrid Pechmann, Cornelia Köhler, Veronka Horber, Oliver Schwartz, Janbernd Kirschner

ABSTRACT

BackgroundSpinal muscular atrophy (SMA) is a severe, life-limiting neurodegenerative disease. A disease-modifying and approved therapy with nusinersen has been available in Germany since July 2017. Gene therapies offer another promising treatment option through a once in a lifetime administration. In May 2019 a gene replacement therapy for the treatment of SMA was approved for the first time by the U.S. Food and Drug Administration (FDA). An application for approval in Europe has been submitted and is currently pending.ObjectiveThis consensus paper was compiled at the invitation of the German Society for Muscular Diseases (DGM) with the participation of all potential German neuromuscular treatment centers, the German section of the Society for Pediatric Neurology (GNP) and with the involvement of the medical scientific advisory board of the DGM. The aim was to define and establish the necessary prerequisites for a safe and successful application of the new gene replacement therapy in clinical practice.ConclusionGene replacement therapy with onasemnogene abeparvovec has the potential to significantly influence the course of SMA. Long-term data on sustainability of effects and possible adverse effects of gene replacement therapy are not yet available. The application of this innovative therapy must be carried out in specialized and appropriately qualified treatment centers under strict safety conditions. This article makes suggestions for the necessary framework conditions and gives recommendations for a systematic pretreatment and posttreatment assessment schedule under gene therapy. The effectiveness and safety of the therapy should be systematically documented in an industry-independent and disease-specific register. More... »

PAGES

518-529

Journal

TITLE

Der Nervenarzt

ISSUE

6

VOLUME

91

Author Affiliations

  • Zentrum für Kinder- und Jugendmedizin Heidelberg, Sektion Neuropädiatrie und Stoffwechselmedizin, Universitätsklinikum Heidelberg, Im Neuenheimer Feld 430, 69120, Heidelberg, Deutschland
  • Abteilung Neuropädiatrie und Sozialpädiatrie, Klinik für Kinder- und Jugendmedizin, Universitätsmedizin Göttingen, Göttingen, Deutschland
  • Abteilung für Neuropädiatrie, Zentrum für neuromuskuläre Erkrankungen des Kindes- und Jugendalters, Universitätsklinikum Essen, Universität Duisburg-Essen, Essen, Deutschland
  • Abteilung Kinderneurologie, Sozialpädiatrie und Epileptologie, Zentrum Kinderheilkunde, Justus-Liebig-Universität, Gießen, Deutschland
  • Dr. v. Hauner’sche Kinderklinik, Universitätskinderklinik, Ludwig-Maximilians-Universität, München, Deutschland
  • Neuropädiatrie, Klinik und Poliklinik für Kinder- und Jugendmedizin, Universitätsklinikum Hamburg-Eppendorf, Hamburg, Deutschland
  • Abteilung Neuropädiatrie, Medizinische Fakultät Carl Gustav Carus, Technische Universität Dresden, Dresden, Deutschland
  • Klinik für Kinder- und Jugendmedizin, DRK Kliniken Berlin, Berlin, Deutschland
  • Sozialpädiatrisches Zentrum, Charité – Universitätsmedizin Berlin, Berlin, Deutschland
  • Friedrich-Baur-Institut, Neurologische Klinik und Poliklinik, Ludwig-Maximilians-Universität, München, Deutschland
  • Klinik für Neuropädiatrie, Universitätsklinikum Jena, Jena, Deutschland
  • Klinik für Neuropädiatrie und Muskelerkrankungen, Zentrum für Kinder- und Jugendmedizin, Universitätsklinikum Freiburg, Medizinische Fakultät, Albert-Ludwigs-Universität Freiburg, Freiburg, Deutschland
  • Abteilung für Neuropädiatrie mit Sozialpädiatrie, Klinik für Kinder- und Jugendmedizin, Ruhr-Universität Bochum, Bochum, Deutschland
  • Abteilung Neuropädiatrie, Entwicklungsneurologie und Sozialpädiatrie, Universitätsklinik für Kinder- und Jugendmedizin, Tübingen, Deutschland
  • Abteilung für Allgemeine Pädiatrie – Neuropädiatrie, Klinik für Kinder- und Jugendmedizin, Universitätsklinikum Münster, Münster, Deutschland
  • Abteilung Neuropädiatrie und Sozialpädiatrisches Zentrum, Zentrum für Kinderheilkunde, Universitätsklinikum Bonn, Bonn, Deutschland
  • Identifiers

    URI

    http://scigraph.springernature.com/pub.10.1007/s00115-020-00919-8

    DOI

    http://dx.doi.org/10.1007/s00115-020-00919-8

    DIMENSIONS

    https://app.dimensions.ai/details/publication/pub.1127514887

    PUBMED

    https://www.ncbi.nlm.nih.gov/pubmed/32394004


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