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Article

Drug discovery for X-linked adrenoleukodystrophy: An unbiased screen for compounds that lower very long-chain fatty acids.

Journal of cellular biochemistry - 1 Oct 2021

Moser Ann B, Liu Yanqiu, Shi Xiaohai, Schrifl Ulrike, Hiebler Shandi, Fatemi Ali, Braverman Nancy E, Steinberg Steven J, Watkins Paul A

Abstract excerpt

X-linked adrenoleukodystrophy (XALD) is a genetic neurologic disorder with multiple phenotypic presentations and limited therapeutic options. The childhood cerebral phenotype (CCALD), a fatal demyelinating disorder affecting about 35% of patients, and the adult-onset adrenomyeloneuropathy (AMN), a peripheral neuropathy affecting 40%-45% of patients, are both caused by mutations in the ABCD1 gene. Both phenotypes...

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Identifiers

PMID: 34056752
DOI: 10.1002/jcb.30014
PMCID: PMC8980593

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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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