{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,13]],"date-time":"2026-10-13T23:45:29Z","timestamp":1791935129998,"version":"4.3.4"},"reference-count":0,"publisher":"Georg Thieme Verlag KG","license":[{"start":{"date-parts":[[2026,10,13]],"date-time":"2026-10-13T00:00:00Z","timestamp":1791849600000},"content-version":"vor","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by\/4.0\/"}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":["Neuropediatrics"],"abstract":"<jats:p>Background: Intrathecal (IT) delivery of gene therapy is an emerging strategy for Spinal Muscular Atrophy (SMA), aiming to enhance central nervous system targeting while potentially reducing systemic exposure. The new administration route of Onasemnogene abeparvovec is associated with a distinct safety profile, including dorsal root ganglia toxicity, and requires structured monitoring beyond existing intravenous guidelines, including planning for long-term multidisciplinary follow-up in pediatric and adult care as well as structured transition protocols.\nMethods:We expanded the existing intravenous monitoring guideline by incorporating surveillance for potential dorsal root ganglia-related adverse effects. The protocol integrates clinical, laboratory, electrophysiological, imaging, and long-term follow-up recommendations.\nResults:The protocol defines pre-treatment assessment, peri-procedural management, and longitudinal follow-up in both pediatric and adult care settings, with emphasis on proactive evaluation of dorsal root ganglia function and structured transition of adolescents treated before adulthood to specialized multidisciplinary adult neurology services.\nConclusions:A structured, multimodal monitoring approach-including dorsal root ganglia surveillance, multidisciplinary long-term follow-up, and coordinated transition from pediatric to adult care-is essential for the safe implementation of intrathecal Onasemnogene abeparvovec in SMA.<\/jats:p>","DOI":"10.1055\/a-2976-6524","type":"journal-article","created":{"date-parts":[[2026,10,13]],"date-time":"2026-10-13T23:02:53Z","timestamp":1791932573000},"source":"Crossref","is-referenced-by-count":0,"title":["Consensus on the use of intrathecal gene therapy for spinal muscular atrophy"],"prefix":"10.1055","author":[{"given":"Astrid","family":"Blaschek","sequence":"additional","affiliation":[{"name":"Dr. von Hauner Children\u2019s Hospital, LMU University Hospital, LMU Medizin, Ludwigs-Maximilians-Universit\u00e4t M\u00fcnchen, Germany","place":["Munich"],"department":["Division of Pediatric Neurology, Developmental Medicine and Social Pediatrics, Department of Pediatrics"]}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Ulrike","family":"Schara-Schmidt","sequence":"additional","affiliation":[{"name":"University Duisburg-Essen, Germany","place":["Essen"],"department":["Dept of Pediatric Neurology, Center for Neuromuscular Disorders in Children and adolescents, Children\u2018s University Hospital"]}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Susanne","family":"Petri","sequence":"additional","affiliation":[{"name":"Hannover Medical School, NDS, Germany","place":["Hanover"],"department":["Department of Neurology"]}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Maggie Christine","family":"Walter","sequence":"additional","affiliation":[{"name":"LMU Klinikum Friedrich-Baur-Institut, BY, Germany","place":["Munich"],"department":["Friedrich-Baur-Institute, Department of Neurology, Ludwig-Maximilians-University of Munich"]}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-6167-5878","authenticated-orcid":false,"given":"Claudia","family":"Wei\u00df","sequence":"additional","affiliation":[{"name":"Charite Universitatsmedizin Berlin, Germany","place":["Berlin"],"department":["Department of Pediatric Neurology"]}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-1925-7538","authenticated-orcid":false,"given":"Katharina","family":"Vill","sequence":"additional","affiliation":[{"name":"Dr. von Hauner Children\u2019s Hospital, LMU University Hospital, LMU Medizin, Ludwigs-Maximilians-Universit\u00e4t M\u00fcnchen, Germany","place":["Munich"],"department":["Division of Pediatric Neurology, Developmental Medicine and Social Pediatrics, Department of Pediatrics"]}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Matthias","family":"Baumann","sequence":"additional","affiliation":[{"name":"Medical University of Innsbruck, Tyrol, Austria","place":["Innsbruck"],"department":["Division of Pediatric Neurology"]}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"G\u00fcnther","family":"Bernert","sequence":"additional","affiliation":[{"name":"Klinik Favoriten, Austria","place":["Vienna"],"department":["Department of Paediatrics"]}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-7696-4808","authenticated-orcid":false,"given":"Marina","family":"Flotats Bastardas","sequence":"additional","affiliation":[{"name":"Saarland University, SL, Germany","place":["Saarbr\u00fccken"],"department":["Department of General Pediatrics and Neonatology, Division of Neuropediatrics"]}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Maja","family":"Von Der Hagen","sequence":"additional","affiliation":[{"name":"Technische Universit\u00e4t Dresden, Germany","place":["Dresden"],"department":["Department of Neuropediatrics, Medizinische Fakult\u00e4t Carl Gustav Carus"]}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jessika","family":"Johannsen","sequence":"additional","affiliation":[{"name":"University Medical Center Hamburg-Eppendorf, Germany","place":["Hamburg"],"department":["Department of Paediatrics"]}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Cornelia","family":"K\u00f6hler","sequence":"additional","affiliation":[{"name":"University Hospital Ruhr-University Bochum, Germany","place":["Bochum"],"department":["Children`s Hospital St. Josef"]}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-0906-8816","authenticated-orcid":false,"given":"Georg M. M.","family":"Stettner","sequence":"additional","affiliation":[{"name":"University of Zurich, ZH, Switzerland","place":["Z\u00fcrich"],"department":["Neuromuscular Center Z\u00fcrich and Department of Pediatric Neurology, Children`s Hospital Z\u00fcrich"]}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Janbernd","family":"Kirschner","sequence":"additional","affiliation":[{"name":"University of Freiburg Faculty of Medicine, BW, Germany","place":["Freiburg"],"department":["Department of Neuropediatrics and Muscle Disorders, Medical Center"]}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Astrid","family":"Pechmann","sequence":"additional","affiliation":[{"name":"University of Freiburg Faculty of Medicine, BW, Germany","place":["Freiburg"],"department":["Department of Neuropediatrics and Muscle Disorders, Medical Center"]}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Andreas","family":"Hahn","sequence":"additional","affiliation":[{"name":"University Hospital, Feulgenstr, Germany","place":["Giessen"],"department":["Department of Child Neurology and Center for Rare Diseases"]}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-8942-7662","authenticated-orcid":false,"given":"Markus","family":"Weiler","sequence":"additional","affiliation":[{"name":"University Hospital Heidelberg, BW, Germany","place":["Heidelberg"],"department":["Department of Neurology"]}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Tim","family":"Hagenacker","sequence":"additional","affiliation":[{"name":"University of Duisburg-Essen, NRW, Germany","place":["Duisburg"],"department":["Department of Neurology"]}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-7574-1231","authenticated-orcid":false,"given":"Andreas","family":"Ziegler","sequence":"additional","affiliation":[{"name":"Heidelberg University, Germany","place":["Heidelberg"],"department":["Medical Faculty Heidelberg, Center for Pediatric and Adolescent Medicine, Department I, Division of Pediatric Neurology and Metabolic Medicine"]}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"194","container-title":["Neuropediatrics"],"original-title":[],"language":"en","link":[{"URL":"http:\/\/www.thieme-connect.de\/products\/ejournals\/pdf\/10.1055\/a-2976-6524.pdf","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2026,10,13]],"date-time":"2026-10-13T23:02:53Z","timestamp":1791932573000},"score":1,"resource":{"primary":{"URL":"http:\/\/www.thieme-connect.de\/DOI\/DOI?10.1055\/a-2976-6524"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2026,10,13]]},"references-count":0,"URL":"https:\/\/doi.org\/10.1055\/a-2976-6524","archive":["Portico","CLOCKSS"],"relation":{},"ISSN":["0174-304X","1439-1899"],"issn-type":[{"value":"0174-304X","type":"print"},{"value":"1439-1899","type":"electronic"}],"subject":[],"published-other":{"date-parts":[[2026,10,13]]},"published":{"date-parts":[[2026,10,13]]}}}