GLYCERYL TRIOLEATE AND TRIERUCATE THERAPY OF ADRENOLEUKODYSTROPHY

三油酸甘油酯和三芥酸酯治疗肾上腺脑白质营养不良

• 批准号: 3765154
• 负责人: HUGO W MOSER
• 金额: --
• 依托单位: JOHNS HOPKINS UNIVERSITY
• 依托单位国家: 美国
• 资助国家: 美国
• 起止时间: 至
• 项目状态: 未结题
• 来源: https://reporter.nih.gov/project-details/3765154
• 关键词: adrenoleukodystrophy; diet therapy; dietary lipid; dietary supplements; human subject; human therapy evaluation

Administration of glyceryl trioleate and trierucate oils has been shown to correct the principal biochemical abnormality in adrenoleukodystrophy (ALD), namely the abnormal accumulation of saturated very long chain fatty acids. Studies are in progress in 366 patients who represent the three major phenotypes of ALD.

三油酸甘油酯和三芥酸酯油的给药已显示 纠正肾上腺脑白质营养不良的主要生化异常 (ALD)即饱和超长链的异常积累 脂肪酸 研究正在366名患者中进行，这些患者代表了 ALD的三种主要表型。