课题基金 / 基金详情

ENZYME REPLACEMENT THERAPY FOR PATIENT W/ MUCOPOLYSACCHARIDOSIS I

ENZYME REPLACEMENT THERAPY FOR PATIENT W/ MUCOPOLYSACCHARIDOSIS I
粘多糖症 I 型患者的酶替代疗法
批准号：
6303934
负责人：
JOSEPH J. MUENZER
金额：
$0.02万
依托单位：
UNIV OF NORTH CAROLINA CHAPEL HILL
依托单位国家：
美国
项目类别：
财政年份：
1999
资助国家：
美国
项目状态：
已结题
起止时间：
1999-12-01 至 2000-11-30
来源：
https://reporter.nih.gov/project-details/6303934
关键词：
O glycosidase child (0-11) clinical research enzyme therapy human subject human therapy evaluation mucopolysaccharidosis type I

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中文摘要
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英文摘要
The purpose of this research study is to determine whether replacing the enzyme, iduronidase, which is missing in children with Mucopolysaccharidosis I (MPS I), is a safe procedure and can help slow or stop the disease. An enzyme is a type of tool used to construct the body as well as recycle and repair it. MPS children are missing an enzyme from their body and therefore, cannot recycle certain body components normally. Iduronidase, a new ivestigational product, is much like that of a normal human enzyme and should be a sufficient substitute.
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