Evidence map›Paper›PMID 42127456›Full record

ArticleNeurotherapeutics : the journal of the American Society for Experimental NeuroTherapeutics2026

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

Natalie Y Lim, Christian E Meagher, Ann Bye, Michelle Lorentzos, Russell C Dale, Shekeeb Mohammad, Katherine B Howell, Elizabeth E Palmer, Ian R Woodcock, Emma Macdonald-Laurs and 9 more

Abstract readConsensus StatementScoping Review
PubMed Full text at PMC Publisher
In one paragraph

Article in Neurotherapeutics : the journal of the American Society for Experimental NeuroTherapeutics, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 1 paper.

0numbers the graph read from it
0cells of the map it votes in
1citing papers in PubMed
–field-weighted citation impact
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

2 · The registry

The trial behind it

Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.

Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.

3 · Its place in the literature

Who cites it

1 citing paper in PubMed.

  1. Review
4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

19 authors.

Natalie Y LimDepartment of Neurology, Sydney Children's Hospital, Sydney, New South Wales, Australia. Electronic address: natalie.lim@health.nsw.gov.au.
Christian E MeagherDiscipline of Paediatrics and Child Health, School of Clinical Medicine, University of New South Wales, Sydney, New South Wales, Australia.
Ann ByeDepartment 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.
Michelle LorentzosSydney 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.
Russell C DaleSchool 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.
Shekeeb MohammadSchool 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.
Katherine B HowellDepartment 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.
Elizabeth E PalmerDiscipline 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.
Ian R WoodcockDepartment 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.
Emma Macdonald-LaursDepartment 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.
Anne PreiszSydney 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.
Nadia BadawiCerebral 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.
Sandra T CooperSchool 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.
Stephen C HilleFOXG1 Research Foundation, Australia.
Toby N TrahairDiscipline 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.
Anita CairnsNeurosciences Department, Queensland Children's Hospital, South Brisbane, Queensland, Australia.
Nicholas SmithDepartment 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.
Michelle A FarrarDepartment 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.
Didu S KariyawasamDepartment 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.

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

Advanced therapies (ATs), including gene and stem cell therapy, hold great potential for preventing and ameliorating many rare neurological disorders (RNDs) in children. These technologies are set to expand across modalities, potentially disrupting and augmenting conventional therapeutic pipelines, with the rapid pace of development highlighting data gaps and implementational challenges. We conducted a two-round modified Delphi study to co-develop a practice framework supporting the safe and effective application of advanced and/or experimental neurotherapeutics for children with rare neurological disorders within a public health ecosystem. The study generated 101 consensus recommendations encompassing criteria to 1) facilitate equitable and timely therapeutic access, 2) optimise transparent communication and shared decision making with families, 3) incorporate disease and patient level considerations for minimising risk and optimising safety within advanced therapeutic research, 4) strengthen resourcing of health systems to enable longitudinal evaluation of treatment effects and safety. Embedding this framework into practice will depend on enhancement of workforce training, establishment of digital infrastructure, fit-for-purpose clinical environments and education and engagement of patients, families and the broader community.

Indexed as

Genetic TherapyNervous System DiseasesPediatricsRare DiseasesTranslational Research, BiomedicalChildHumansStem Cell TransplantationImplementationIndividualized medicineNeurologic disordersPrecision medicineTranslation

Identifiers

PMID42127456
PMCPMC13195611

What OpenQuestion holds

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Registered trials

None linked

Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.