An alternative therapeutic approach to haematopoetic stem cell transplantation in early cerebral adrenoleukodystrophy
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This scientific commentary refers to ‘Leriglitazone halts disease progression in adult patients with early cerebral adrenoleukodystrophy’ by Golse et al. (https://doi.org/10.1093/brain/awae169).
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Initial results suggested that leriglitazone could halt neuroinflammation and disease progression up to 2 years in adult patients with early cALD.
“The initial results suggested that leriglitazone could halt neuroinflammation and disease progression up to 2 years [466,467].”
From Revisiting the Pathogenesis of X-Linked Adrenoleukodystrophy (2025) · cites this paper as [467]
Section 2 (p. 2) says the small adult early-cALD cohort suggests leriglitazone can halt neuroinflammation and disease progression. It does not give a two-year duration for that finding; the 96-week trial it mentions involved patients with AMN, not the adult early-cALD cohort. (LLM, conf=0.86)
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Cites
Kühl, J.-S., Suarez, F., Gillett, G. T., Hemmati, P. G., Snowden, J. A., Stadler, M., Vuong, G. L., Aubourg, P., Köhler, W., & Arnold, R. (2017) · Brain · doi:10.1093/brain/awx016 · Cited by 78 · see citing works
Köhler, W., Engelen, M., Eichler, F., Lachmann, R., Fatemi, A., Sampson, J., Salsano, E., Gamez, J., Molnar, M. J., Pascual, S., et al. (2023) · The Lancet Neurology · doi:10.1016/s1474-4422(22)00495-1 · Cited by 48 · see citing works
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Research works printed but not linked: 5
- de Beer M, Engelen M, van Geel BM. Frequent occurrence of cerebral demyelination in adrenomyeloneuropathy. Neurology. 2014;83:2227-2231.
- Huffnagel IC, Laheji FK, Aziz-Bose R, et al. The Natural History of Adrenal Insufficiency in X-Linked Adrenoleukodystrophy: An International Collaboration. J Clin Endocrinol Metab. 2019;104:118-126.
- Huffnagel IC, van Ballegoij WJC, van Geel BM, Vos JMBW, Kemp S, Engelen M. Progression of myelopathy in males with adrenoleukodystrophy: towards clinical trial readiness. Brain. 2019;142:334-343.
- Golse M, Weinhofer I, Blanco B, et al. Leriglitazone: an alternative therapeutic approach to HSCT in early cerebral adrenoleukodystrophy. Brain. Published online 4 June 2024. https://doi.org/10.1093/brain/awae169 · DOI · Paper page
- Schöls L. Leriglitazone: frustration and hope in adrenoleukodystrophy. Lancet Neurol. 2023;22:103-105.
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Revisiting the Pathogenesis of X-Linked Adrenoleukodystrophy
Bougnères, P., & Le Stunff, C. (2025) · Genes · doi:10.3390/genes16050590 · Cited by 11 · see citing works
- Authors
- Chataway, J., Wade, C., Murphy, E., & Lynch, D. S.
- Affiliations
- University College London Hospitals NHS Foundation Trust (GB)
- Corresponding author
- Chataway, Jeremy
- Venue
- Brain
- Publisher
- Oxford University Press (OUP)
- Published
- 2024-08-07
- Volume
- 147
- Issue
- 10
- Pages
- 3271-3273
- ISSN
- 0006-8950 (ISSN-L)
- 1460-2156
- OpenAlex
- W4401439334
- Cited by
- 1
- References
- 2
- Citations by year
- 2025: 1
- Type
- journal article
- Language
- en
- Open access
- green
- discovery.ucl.ac.uk
- Locations
- Brain (publishedVersion)
- PubMed (publishedVersion)
- UCL Discovery (University College London) (submittedVersion)
- Topics
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Clinical Biochemistry › Metabolism and Genetic Disorders
- Health Sciences › Medicine › Physiology › Lysosomal Storage Disorders Research
- Health Sciences › Medicine › Pathology and Forensic Medicine › Multiple Sclerosis Research Studies
- Keywords
- cerebral adrenoleukodystrophy, hematopoietic stem cell transplantation, disease progression
- MeSH terms
- Adrenoleukodystrophy, Adult, Aged, Humans, Male, Middle Aged, Research Design, Randomized Controlled Trials as Topic, Hematopoietic Stem Cell Transplantation, Young Adult
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