A model-based approach to assess the exposure-response relationship of Lorenzo's oil in adrenoleukodystrophy
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- 作者：Mariam A. Ahmed ; Reena V. Kartha ; Richard C. Brundage ; James Cloyd ; Cynthia Basu ; Bradley P. Carlin ; Richard O. Jones ; Ann B. Moser ; Ali Fatemi and Gerald V. Raymond
- 刊名：British Journal of Clinical Pharmacology
- 出版年：2016
- 出版时间：June 2016
- 年：2016
- 卷：81
- 期：6
- 页码：1058-1066
- 全文大小：516K
- ISSN：1365-2125
文摘
X-linked adrenoleukodystrophy (X-ALD) is a peroxisomal disorder, most commonly affecting boys, associated with increased very long chain fatty acids (C26:0) in all tissues, causing cerebral demyelination and adrenocortical insufficiency. Certain monounsaturated long chain fatty acids including oleic and erucic acids, known as Lorenzo's oil (LO), lower plasma C26:0 levels. The aims of this study were to characterize the effect of LO administration on plasma C26:0 concentrations and to determine whether there is an association between plasma concentrations of erucic acid or C26:0 and the likelihood of developing brain MRI abnormalities in asymptomatic boys.