{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,13]],"date-time":"2026-10-13T02:50:46Z","timestamp":1791859846904,"version":"4.3.4"},"reference-count":27,"publisher":"Oxford University Press (OUP)","issue":"10","license":[{"start":{"date-parts":[[2024,6,4]],"date-time":"2024-06-04T00:00:00Z","timestamp":1717459200000},"content-version":"vor","delay-in-days":0,"URL":"https:\/\/academic.oup.com\/pages\/standard-publication-reuse-rights"}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":[],"published-print":{"date-parts":[[2024,10,3]]},"abstract":"<jats:title>Abstract<\/jats:title>\n                  <jats:p>Cerebral adrenoleukodystrophy (CALD) is an X-linked rapidly progressive demyelinating disease leading to death usually within a few years. The standard of care is haematopoietic stem cell transplantation (HSCT), but many men are not eligible due to age, absence of a matched donor or lesions of the corticospinal tracts (CST). Based on the ADVANCE study showing that leriglitazone decreases the occurrence of CALD, we treated 13 adult CALD patients (19\u201367 years of age) either not eligible for HSCT (n = 8) or awaiting HSCT (n = 5).<\/jats:p>\n                  <jats:p>Patients were monitored every 3 months with standardized neurological scores, plasma biomarkers and brain MRI comprising lesion volumetrics and diffusion tensor imaging.<\/jats:p>\n                  <jats:p>The disease stabilized clinically and radiologically in 10 patients with up to 2 years of follow-up. Five patients presented with gadolinium enhancing CST lesions that all turned gadolinium negative and, remarkably, regressed in four patients. Plasma neurofilament light chain levels stabilized in all 10 patients and correlated with lesion load. The two patients who continued to deteriorate were over 60 years of age with prominent cognitive impairment. One patient died rapidly from coronavirus disease 2019.<\/jats:p>\n                  <jats:p>These results suggest that leriglitazone can arrest disease progression in adults with early-stage CALD and may be an alternative treatment to HSCT.<\/jats:p>","DOI":"10.1093\/brain\/awae169","type":"journal-article","created":{"date-parts":[[2024,5,31]],"date-time":"2024-05-31T20:16:19Z","timestamp":1717186579000},"page":"3344-3351","source":"Crossref","is-referenced-by-count":24,"title":["Leriglitazone halts disease progression in adult patients with early cerebral adrenoleukodystrophy"],"prefix":"10.1093","volume":"147","author":[{"given":"Marianne","family":"Golse","sequence":"first","affiliation":[{"name":"Sorbonne Universit\u00e9, INSERM, CNRS, Laboratoire d'Imagerie Biom\u00e9dicale , 75013 Paris ,","place":["France"]},{"name":"Department of Neuroradiology, AP-HP, Piti\u00e9-Salp\u00eatri\u00e8re University Hospital , 75013 Paris ,","place":["France"]}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Isabelle","family":"Weinhofer","sequence":"additional","affiliation":[{"name":"Department of Pathobiology of the Nervous System, Center for Brain Research, Medical University of Vienna , 1090 Vienna ,","place":["Austria"]}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Bernardo","family":"Blanco","sequence":"additional","affiliation":[{"name":"Department of Medical Genetics, Reference Centers for Adult Neurometabolic diseases and Adult Leukodystrophies, AP-HP, Piti\u00e9-Salp\u00eatri\u00e8re University Hospital , 75013 Paris ,","place":["France"]},{"name":"INSERM U 1127, CNRS UMR 7225, Sorbonne Universit\u00e9s, UPMC Univ Paris 06 UMR S 1127, Institut du Cerveau, ICM , 75013 Paris ,","place":["France"]}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Magali","family":"Barbier","sequence":"additional","affiliation":[{"name":"INSERM U 1127, CNRS UMR 7225, Sorbonne Universit\u00e9s, UPMC Univ Paris 06 UMR S 1127, Institut du Cerveau, ICM , 75013 Paris ,","place":["France"]}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Elise","family":"Yazbeck","sequence":"additional","affiliation":[{"name":"Department of Medical Genetics, Reference Centers for Adult Neurometabolic diseases and Adult Leukodystrophies, AP-HP, Piti\u00e9-Salp\u00eatri\u00e8re University Hospital , 75013 Paris ,","place":["France"]}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Camille","family":"Huiban","sequence":"additional","affiliation":[{"name":"INSERM U 1127, CNRS UMR 7225, Sorbonne Universit\u00e9s, UPMC Univ Paris 06 UMR S 1127, Institut du Cerveau, ICM , 75013 Paris ,","place":["France"]}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Boris","family":"Chaumette","sequence":"additional","affiliation":[{"name":"GHU Paris Psychiatrie & Neurosciences, Saint-Anne Hospital , 75014 Paris ,","place":["France"]}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Bertrand","family":"Pichon","sequence":"additional","affiliation":[{"name":"Department of Neurology, AP-HP, Piti\u00e9-Salp\u00eatri\u00e8re University Hospital , 75013 Paris ,","place":["France"]}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Ali","family":"Fatemi","sequence":"additional","affiliation":[{"name":"Moser Center for Leukodystrophies, Kennedy Krieger Institute and Department of Neurology, Johns Hopkins University , Baltimore, MD 21205 ,","place":["USA"]}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Silvia","family":"Pascual","sequence":"additional","affiliation":[{"name":"Minoryx Therapeutics , 08302 Barcelona ,","place":["Spain"]}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Marc","family":"Martinell","sequence":"additional","affiliation":[{"name":"Minoryx Therapeutics , 08302 Barcelona ,","place":["Spain"]}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Johannes","family":"Berger","sequence":"additional","affiliation":[{"name":"Department of Pathobiology of the Nervous System, Center for Brain Research, Medical University of Vienna , 1090 Vienna ,","place":["Austria"]}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Vincent","family":"Perlbarg","sequence":"additional","affiliation":[{"name":"BrainTale , 75013 Paris ,","place":["France"]}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Damien","family":"Galanaud","sequence":"additional","affiliation":[{"name":"Sorbonne Universit\u00e9, INSERM, CNRS, Laboratoire d'Imagerie Biom\u00e9dicale , 75013 Paris ,","place":["France"]},{"name":"Department of Neuroradiology, AP-HP, Piti\u00e9-Salp\u00eatri\u00e8re University Hospital , 75013 Paris ,","place":["France"]}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Fanny","family":"Mochel","sequence":"additional","affiliation":[{"name":"Department of Medical Genetics, Reference Centers for Adult Neurometabolic diseases and Adult Leukodystrophies, AP-HP, Piti\u00e9-Salp\u00eatri\u00e8re University Hospital , 75013 Paris ,","place":["France"]},{"name":"INSERM U 1127, CNRS UMR 7225, Sorbonne Universit\u00e9s, UPMC Univ Paris 06 UMR S 1127, Institut du Cerveau, ICM , 75013 Paris ,","place":["France"]}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"286","published-online":{"date-parts":[[2024,6,4]]},"reference":[{"key":"2024100318031135700_awae169-B1","doi-asserted-by":"crossref","first-page":"1485","DOI":"10.1093\/brain\/120.8.1485","article-title":"Adrenoleukodystrophy: Phenotype, genetics, pathogenesis and 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Systematic review, qualitative assessment, and meta-analysis of studies validating microstructural imaging with myelin histology","volume":"230","author":"Lazari","year":"2021","journal-title":"Neuroimage"},{"key":"2024100318031135700_awae169-B12","doi-asserted-by":"crossref","first-page":"102566","DOI":"10.1016\/j.nicl.2021.102566","article-title":"Multiparametric in vivo analyses of the brain and spine identify structural and metabolic biomarkers in men with adrenomyeloneuropathy","volume":"29","author":"Adanyeguh","year":"2021","journal-title":"Neuroimage Clin"},{"key":"2024100318031135700_awae169-B13","doi-asserted-by":"crossref","first-page":"1735","DOI":"10.1093\/brain\/aww068","article-title":"Quantitative MRI of the spinal cord and brain in adrenomyeloneuropathy: In vivo assessment of structural 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