GLYCERYL TRIERUCATE THERAPY FOR ADRENOLEUKODYSTROPHY

三聚甘油酯治疗肾上腺脑白质营养不良

• 批准号: 6245313
• 负责人: HUGO W MOSER
• 金额: $ 2.23万
• 依托单位: JOHNS HOPKINS UNIVERSITY
• 依托单位国家: 美国
• 财政年份: 1997
• 资助国家: 美国
• 起止时间: 1997-03-05 至 1997-11-30
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/6245313
• 关键词: adrenoleukodystrophy; brain imaging /visualization /scanning; child (0-11); clinical research; diet therapy; endocrine disorder chemotherapy; human subject; human therapy evaluation; long chain fatty acid; longitudinal human study; magnetic resonance imaging; male; nutrition related tag; pediatric pharmacology; triglycerides

GTE therapy is offered to all boys with the biochemical defect of ALD.This therapy reverses the principal biochemical abnormality, namely, the accumulation of very long chain fatty acids.We are encouraged by the fact that all the pts. who were neurologically symptomatic at the beginning of therapy have remained so & that their brain MRI studies also have remained stable. Mean duration of therapy is 14 months. Longer follow-up is required for definitive conclusion.

GTE疗法适用于所有患有爱滋病生化缺陷的男孩。 治疗逆转了主要的生化异常，即 超长链脂肪酸的积累。我们对这一事实感到鼓舞 所有的点数。他们在一开始就有神经学症状 治疗一直如此，他们的脑部核磁共振研究也保持不变 稳定。平均疗程14个月。更长的随访时间是 需要有明确的结论。