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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
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  Data: Strategic consensus on the clinical translation of advanced therapies in paediatric rare neurological disorders.
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  Data: <searchLink fieldCode="AU" term="%22Lim+NY%22">Lim NY</searchLink>; Department of Neurology, Sydney Children's Hospital, Sydney, New South Wales, Australia. Electronic address: natalie.lim@health.nsw.gov.au.<br /><searchLink fieldCode="AU" term="%22Meagher+CE%22">Meagher CE</searchLink>; Discipline of Paediatrics and Child Health, School of Clinical Medicine, University of New South Wales, Sydney, New South Wales, Australia.<br /><searchLink fieldCode="AU" term="%22Bye+A%22">Bye A</searchLink>; 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.<br /><searchLink fieldCode="AU" term="%22Lorentzos+M%22">Lorentzos M</searchLink>; 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.<br /><searchLink fieldCode="AU" term="%22Dale+RC%22">Dale RC</searchLink>; 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.<br /><searchLink fieldCode="AU" term="%22Mohammad+S%22">Mohammad S</searchLink>; 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.<br /><searchLink fieldCode="AU" term="%22Howell+KB%22">Howell KB</searchLink>; 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.<br /><searchLink fieldCode="AU" term="%22Palmer+EE%22">Palmer EE</searchLink>; 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.<br /><searchLink fieldCode="AU" term="%22Woodcock+IR%22">Woodcock IR</searchLink>; 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.<br /><searchLink fieldCode="AU" term="%22Macdonald-Laurs+E%22">Macdonald-Laurs E</searchLink>; 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.<br /><searchLink fieldCode="AU" term="%22Preisz+A%22">Preisz A</searchLink>; 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.<br /><searchLink fieldCode="AU" term="%22Badawi+N%22">Badawi N</searchLink>; 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.<br /><searchLink fieldCode="AU" term="%22Cooper+ST%22">Cooper ST</searchLink>; 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.<br /><searchLink fieldCode="AU" term="%22Hille+SC%22">Hille SC</searchLink>; FOXG1 Research Foundation, Australia.<br /><searchLink fieldCode="AU" term="%22Trahair+TN%22">Trahair TN</searchLink>; 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.<br /><searchLink fieldCode="AU" term="%22Cairns+A%22">Cairns A</searchLink>; Neurosciences Department, Queensland Children's Hospital, South Brisbane, Queensland, Australia.<br /><searchLink fieldCode="AU" term="%22Smith+N%22">Smith N</searchLink>; 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.<br /><searchLink fieldCode="AU" term="%22Farrar+MA%22">Farrar MA</searchLink>; 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.<br /><searchLink fieldCode="AU" term="%22Kariyawasam+DS%22">Kariyawasam DS</searchLink>; 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.
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  Data: <searchLink fieldCode="JN" term="%22101290381%22">Neurotherapeutics : the journal of the American Society for Experimental NeuroTherapeutics</searchLink> [Neurotherapeutics] 2026 Apr; Vol. 23 (3), pp. e00921. <i>Date of Electronic Publication: </i>2026 May 12.
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