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Article

Molecular Biomarkers for Adrenoleukodystrophy: An Unmet Need

6 Dec 2021

Abstract excerpt

gene and the accumulation of very long-chain fatty acids in plasma and tissues. Patients present with heterogeneous clinical manifestations which can include adrenal insufficiency, myelopathy, and/or cerebral demyelination. In the absence of a genotype-phenotype correlation, the clinical outcome of an individual cannot be predicted and currently there are no molecular markers available to quantify disease...

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PMID: 34943935
DOI: 10.3390/cells10123427

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