Presymptomatic Lesion in Childhood Cerebral Adrenoleukodystrophy: Timing and Treatment.
Sign Up to like & getrecommendations! 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… read more here.
Keywords: ccald; childhood; cerebral adrenoleukodystrophy; lesion ... See more keywords
Isoprostanoid Plasma Levels Are Relevant to Cerebral Adrenoleukodystrophy Disease
Sign Up to like & getrecommendations! 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… read more here.
Keywords: plasma levels; cerebral disease; cerebral adrenoleukodystrophy; disease ... See more keywords