Article

Pharmacological complementation remedies an inborn error of lipid metabolism

2019-11-20

Abstract excerpt

<h4>Summary</h4> X-linked adrenoleukodystrophy (X-ALD) is a rare, genetic disease in which increased very long chain fatty acids (VLCFAs) in the central nervous system (CNS) cause demyelination and axonal degeneration, leading to severe neurological deficits. Sobetirome, a potent thyroid hormone agonist, has been shown to lower VLCFA levels in the periphery and CNS. In this study, two pharmacological strategies f...

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Literature Corpus work: bdaa98aa-c30a-570e-b20d-9815a00fdb8d
DOI: 10.1101/848119

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