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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：
6297262
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：
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/6297262
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 study is to test the safety and efficacy of enzyme (recombinant human a-L-iduronidase) replacement in patients with mucopolysaccharidosis I (MPS I). This multi-center clinical study (phase II-III) is directed by Dr. Emil Kakkis, who recruited the patients and directs the enyzme production in the Iduronidase Production Laboratory, Torrance, CA. BioMarin Pharmaceutical, Inc. have been assigned the Investigational New Drug Application number BB-IND #7334 for the enzyme to be used in this study. I have been asked to administer at UNC some of the enyzme infusions to two patients with MPS I. Preliminary results have indicated that the enzyme infusions have been beneficial to the patients. Benefits have included decreased liver and spleen size, decreased pain, increased exercise tolerance with decreased joint stiffness, improved gait with better balance, improved vision and hearing.
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