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GLYCERYL TRIERUCATE (GTE) THERAPY FOR ADRENOLEUKODYSTROPHY (ALD)

GLYCERYL TRIERUCATE (GTE) THERAPY FOR ADRENOLEUKODYSTROPHY (ALD)
三聚甘油酯 (GTE) 治疗肾上腺脑白质营养不良 (ALD)
批准号：
6114431
负责人：
HUGO W MOSER
金额：
$2.06万
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/6114431
关键词：
adrenoleukodystrophy 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 outcomes research pediatric pharmacology triglycerides

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中文摘要
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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*
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