Strategic consensus on the clinical translation of advanced therapies in paediatric rare neurological disorders.

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Title: Strategic consensus on the clinical translation of advanced therapies in paediatric rare neurological disorders.
Authors: Lim NY; Department of Neurology, Sydney Children's Hospital, Sydney, New South Wales, Australia. Electronic address: natalie.lim@health.nsw.gov.au., Meagher CE; Discipline of Paediatrics and Child Health, School of Clinical Medicine, University of New South Wales, Sydney, New South Wales, Australia., Bye A; Department of Neurology, Sydney Children's Hospital, Sydney, New South Wales, Australia; Discipline of Paediatrics and Child Health, School of Clinical Medicine, University of New South Wales, Sydney, New South Wales, Australia., Lorentzos M; Sydney Children's Hospitals Network, Sydney, New South Wales, Australia; School of Medical Sciences, Faculty of Medicine and Health, University of Sydney, Sydney, New South Wales, Australia., Dale RC; School of Medical Sciences, Faculty of Medicine and Health, University of Sydney, Sydney, New South Wales, Australia; Kids Neuroscience Centre, Kids Research, The Children's Hospital at Westmead, Sydney, New South Wales, Australia., Mohammad S; School of Medical Sciences, Faculty of Medicine and Health, University of Sydney, Sydney, New South Wales, Australia; Kids Neuroscience Centre, Kids Research, The Children's Hospital at Westmead, Sydney, New South Wales, Australia., Howell KB; Department of Neurology, Royal Children's Hospital, Melbourne, Victoria, Australia; Neuroscience Group, Murdoch Children's Research Institute, Melbourne, Victoria, Australia; Department of Paediatrics, University of Melbourne, Melbourne, Victoria, Australia., Palmer EE; Discipline of Paediatrics and Child Health, School of Clinical Medicine, University of New South Wales, Sydney, New South Wales, Australia; Rare Diseases NSW, Sydney, New South Wales, Australia; Centre for Clinical Genetics, Sydney Children's Hospitals Network, Sydney, New South Wales, Australia., Woodcock IR; Department of Neurology, Royal Children's Hospital, Melbourne, Victoria, Australia; Neuroscience Group, Murdoch Children's Research Institute, Melbourne, Victoria, Australia; Department of Paediatrics, University of Melbourne, Melbourne, Victoria, Australia., Macdonald-Laurs E; Department of Neurology, Royal Children's Hospital, Melbourne, Victoria, Australia; Neuroscience Group, Murdoch Children's Research Institute, Melbourne, Victoria, Australia; Department of Paediatrics, University of Melbourne, Melbourne, Victoria, Australia., Preisz A; Sydney Children's Hospitals Network, Sydney, New South Wales, Australia; School of Medicine, University of Notre Dame Australia, Sydney, New South Wales, Australia; Sydney Health Ethics, University of Sydney, Sydney, New South Wales, Australia., Badawi N; Cerebral Palsy Alliance Research Institute, Specialty of Child and Adolescent Health, Sydney Medical School, The University of Sydney, Sydney, New South Wales, Australia; School of Medical Sciences, Faculty of Medicine and Health, University of Sydney, Sydney, New South Wales, Australia; Grace Centre for Newborn Intensive Care, The Children's Hospital at Westmead, Sydney, New South Wales, Australia., Cooper ST; School of Medical Sciences, Faculty of Medicine and Health, University of Sydney, Sydney, New South Wales, Australia; Kids Neuroscience Centre, Kids Research, The Children's Hospital at Westmead, Sydney, New South Wales, Australia; The Children's Medical Research Institute, Sydney, New South Wales, Australia., Hille SC; FOXG1 Research Foundation, Australia., Trahair TN; Discipline of Paediatrics and Child Health, School of Clinical Medicine, University of New South Wales, Sydney, New South Wales, Australia; Kids Cancer Centre, Sydney Children's Hospital Randwick, Sydney, New South Wales, Australia., Cairns A; Neurosciences Department, Queensland Children's Hospital, South Brisbane, Queensland, Australia., Smith N; Department of Neurology and Clinical Neurophysiology, Women's and Children's Health Network, South Australia, Australia; Discipline of Paediatrics, School of Medicine, University of Adelaide, South Australia, Australia., Farrar MA; Department of Neurology, Sydney Children's Hospital, Sydney, New South Wales, Australia; Discipline of Paediatrics and Child Health, School of Clinical Medicine, University of New South Wales, Sydney, New South Wales, Australia., Kariyawasam DS; Department of Neurology, Sydney Children's Hospital, Sydney, New South Wales, Australia; Discipline of Paediatrics and Child Health, School of Clinical Medicine, University of New South Wales, Sydney, New South Wales, Australia.
Source: Neurotherapeutics : the journal of the American Society for Experimental NeuroTherapeutics [Neurotherapeutics] 2026 Apr; Vol. 23 (3), pp. e00921. Date of Electronic Publication: 2026 May 12.
Publication Type: Consensus Statement; Journal Article; Scoping Review
Journal Info: Publisher: Elsevier Inc. on behalf of American Society for Experimental NeuroTherapeutics Country of Publication: United States NLM ID: 101290381 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1878-7479 (Electronic) Linking ISSN: 18787479 NLM ISO Abbreviation: Neurotherapeutics Subsets: MEDLINE
Database: MEDLINE Ultimate
Description
ISSN:1878-7479
DOI:10.1016/j.neurot.2026.e00921