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Published in 2025 at "Journal of child neurology"
DOI: 10.1177/08830738251353034
Abstract: Objectives More than half of males with X-linked adrenoleukodystrophy (ALD) develop progressive, inflammatory cerebral demyelination (cerebral adrenoleukodystrophy). Treatment for cerebral adrenoleukodystrophy is limited with no standard therapies for advanced cerebral adrenoleukodystrophy. We reviewed cerebral adrenoleukodystrophy…
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Keywords:
immunopathology;
inflammation immunomodulation;
pathology;
adrenoleukodystrophy ... See more keywords
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Published in 2022 at "Neurology"
DOI: 10.1212/wnl.0000000000201440
Abstract: Background and Objectives We sought to characterize the natural history and standard-of-care practices between the radiologic appearance of brain lesions, the appearance of lesional enhancement, and treatment with hematopoietic stem-cell transplant or gene therapy among…
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Keywords:
ccald;
childhood;
cerebral adrenoleukodystrophy;
lesion ... See more keywords
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Published in 2022 at "Life"
DOI: 10.3390/life12020146
Abstract: Cerebral adrenoleukodystrophy (ALD) is a rare neuroinflammatory disorder characterized by progressive demyelination. Mutations within the ABCD1 gene result in very long-chain fatty acid (VLCFA) accumulation within the peroxisome, particularly in the brain. While this VLCFA…
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Keywords:
plasma levels;
cerebral disease;
cerebral adrenoleukodystrophy;
disease ... See more keywords