TREATMENT OF CHOLESTEROL DEFECT IN SMITH LEMLI OPITZ SYNDROME
TREATMENT OF CHOLESTEROL DEFECT IN SMITH LEMLI OPITZ SYNDROME
批准号:
6441957
负责人:
ELLEN ELIAS
金额:
$2.88万
依托单位国家:
美国
项目类别:
财政年份:
2000
资助国家:
美国
项目状态:
已结题
起止时间:
2000-12-01 至 2001-11-30
中文摘要
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英文摘要
Children with Smith-Lemli-Opitz (SLO) Syndrome have a severe cholesterol deficiency, caused by an inborn error of metabolism in the cholesterol synthetic pathway. Children with SLO have multiple birth defects and complex medical problems including severe growth failure, mental retardation, endocrine, dermatologic, hematologic, and behavioral problems. This protocol involves giving pure cholesterol in the form of a concentrated suspension, in an effort to raise cholesterol levels, and lower accumulation of the precurson, 7-dehydrocholesterol. The cholesterol is administered enterally (po or pg). Cholesterol deficiency has effects on multiple organ systems. Patients on protocol will be followed to determine if cholesterol treatment effects growth, development, behavior, hematologic, dermatologic and endocrine status.
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TREATMENT OF CHOLESTEROL DEFECT IN SMITH LEMLI OPITZ SYNDROME
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批准号:6568550
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项目类别:
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资助金额:$2.88万
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财政年份:2001
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负责人:ELLEN ELIAS
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依托单位:
TREATMENT OF CHOLESTEROL DEFECT IN SMITH LEMLI OPITZ SYNDROME
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批准号:6485556
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项目类别:
-
资助金额:$2.88万
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财政年份:2000
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负责人:ELLEN ELIAS
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依托单位:
TREATMENT OF CHOLESTEROL DEFECT IN SMITH LEMLI OPITZ SYNDROME
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批准号:6308958
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项目类别:
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资助金额:$2.88万
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财政年份:1999
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负责人:ELLEN ELIAS
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依托单位:
TREATMENT OF CHOLESTEROL DEFECT IN SMITH LEMLI OPITZ SYNDROME
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批准号:6265632
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项目类别:
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资助金额:$0.07万
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财政年份:1998
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负责人:ELLEN ELIAS
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依托单位:
海外基金