Article
Five men with arresting and relapsing cerebral adrenoleukodystrophy.
Journal of neurology - 1 Mar 2021
Carlson Aaron M, Huffnagel Irene C, Verrips Aad, van der Knaap Marjo S, Engelen Marc, Van Haren Keith
Abstract excerpt
BACKGROUND: X-linked adrenoleukodystrophy (ALD) is the most common genetic peroxisomal disorder with an estimated prevalence of 1:15,000. Approximately two-thirds of males with ALD manifest the inflammatory demyelinating cerebral phenotype (cALD) at some disease stage, in which focal, inflammatory lesions progress over months to years. Hematopoietic stem-cell transplantation can permanently halt cALD progression,...
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