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
翻译
这项研究的目的是确定替换一种名为伊杜糖醛酸酶的酶是否是一种安全的方法，这种酶在患有粘多糖病I （MPS I）的儿童中缺失，可以帮助减缓或阻止这种疾病。酶是一种工具，用于构建身体，以及回收和修复它。MPS儿童体内缺少一种酶，因此不能正常地回收某些身体成分。伊杜糖醛酸酶是一种新的研究产品，它与正常的人体酶非常相似，应该是一种足够的替代品。
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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