课题基金 / 基金详情

EFFECT OF GLYCERYL TRIERUCATE THERAPY ON CLINICAL COURSE OF ADRENOLEUKODYSTROPHY

EFFECT OF GLYCERYL TRIERUCATE THERAPY ON CLINICAL COURSE OF ADRENOLEUKODYSTROPHY
三芥酸甘油酯治疗对肾上腺脑白质营养不良临床病程的影响
批准号：
6218133
负责人：
HUGO W MOSER
金额：
$0.23万
依托单位：
JOHNS HOPKINS UNIVERSITY
依托单位国家：
美国
项目类别：
财政年份：
1998
资助国家：
美国
项目状态：
已结题
起止时间：
1998-12-01 至 1999-11-30
来源：
https://reporter.nih.gov/project-details/6218133
关键词：
adrenoleukodystrophy child (0-11) clinical research clinical trials diet therapy endocrine disorder chemotherapy human subject human therapy evaluation long chain fatty acid longitudinal human study magnetic resonance imaging nutrition related tag outcomes research pathologic process pediatric pharmacology triglycerides

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相关文献

中文摘要
翻译
GTE疗法适用于所有患有ALD生化缺陷的男孩。这种疗法逆转了主要的生化异常，即超长链脂肪酸的积累。我们感到鼓舞的是，所有的患者都是如此。那些在治疗开始时有神经学症状的患者仍然如此&他们的脑部核磁共振研究也保持稳定。平均疗程14个月。需要更长时间的随访才能得出最终结论。
英文摘要
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.
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X-LINKED ADRENOLEUKODYSTROPHY
PLACEBO-CONTROLLED STUDY OF X-ALD DIET THERAPY
  • 批准号：
    7604721
  • 项目类别：
  • 资助金额：
    $0.35万
  • 财政年份：
    2006
  • 负责人：
    HUGO W MOSER
  • 依托单位：
    JOHNS HOPKINS UNIVERSITY
INTERNET MULTICENTER THERAPEUTIC TRIALS OF X LINKED ADRENOLEUKODYSTROPHY
THERAPEUTIC TRIALS OF X-LINKED ALD: PHASE III; LORENZO*