Analysis of Interindividual Lesion Progression Variability in Adult Cerebral Adrenoleukodystrophy.
BACKGROUND AND OBJECTIVES Cerebral adrenoleukodystrophy (cALD), the most severe clinical phenotype of X-linked adrenoleukodystrophy (X-ALD), is characterized by rapidly progressive, inflammatory destruction of the cerebral white matter. In adult patients, therapeutic options are limited to hematopoietic stem cell trans...