ENZYME REPLACEMENT THERAPY IN GAUCHER DISEASE

戈谢病的酶替代疗法

• 批准号: 6307370
• 负责人: Ernest Beutler
• 金额: $ 2.74万
• 依托单位: SCRIPPS RESEARCH INSTITUTE, THE
• 依托单位国家: 美国
• 财政年份: 1999
• 资助国家: 美国
• 起止时间: 1999-12-01 至 2001-02-28
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/6307370
• 关键词: Gaucher's disease; clinical research; dosage; enzyme therapy; glucosylceramidase; human subject; human therapy evaluation; metabolism disorder chemotherapy

This study is designed to determine whether clinically useful effects may be achieved by administering various dosage regimens of glucocerebrosidase to patients with Type I Gaucher Disease, a glycolipid storage disease.

本研究旨在确定对I型戈谢病（一种糖脂蓄积性疾病）患者给予不同剂量方案的葡萄糖脑苷脂酶是否可获得临床有用的效果。