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Management of adrenoleukodystrophy: From pre-clinical studies to the development of new therapies.

Biomedicine & pharmacotherapy = Biomedecine & pharmacotherapie - 1 Nov 2021

Ma Chui Yan, Li Cheng, Zhou Xiaoya, Zhang Zhao, Jiang Hua, Liu Hongsheng, Chen Huanhuan Joyce, Tse Hung-Fat, Liao Can, Lian Qizhou

Abstract excerpt

X-linked adrenoleukodystrophy (X-ALD) is an inherited neurodegenerative disorder associated with mutations of the ABCD1 gene that encodes a peroxisomal transmembrane protein. It results in accumulation of very long chain fatty acids in tissues and body fluid. Along with other factors such as epigenetic and environmental involvement, ABCD1 mutation-provoked disorders can present different phenotypes including...

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PMID: 34560537
DOI: 10.1016/j.biopha.2021.112214

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PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 7 Sept 2026. Manifest publication-graph-cohort-2026-09-08.1.

Source receipt: pubmed, retrieved 7 Sept 2026.

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