Presymptomatic Lesion in Childhood Cerebral Adrenoleukodystrophy: Timing and Treatment.
Eric James MallackKeith P Van HarenAnna TorreyStephanie van de StadtMarc EngelenGerald V RaymondAli FatemiFlorian S EichlerPublished in: Neurology (2022)
Our findings offer a more refined understanding of the timing of lesion formation, enhancement, and treatment among boys with presymptomatic CCALD. These data offer benchmarks for standardizing clinical care and designing future clinical trials.