Article

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

2024-01-30

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 cereb...

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Literature Corpus work: 8442b858-a693-5ea7-9c8c-ef51c85b5c64
DOI: 10.22541/au.170665141.13562390/v1

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