TREATMENT OF UREA CYCLE ENZYMOPATHIES

尿素循环酶病的治疗

• 批准号: 6218132
• 负责人: SAUL W BRUSILOW
• 金额: $ 0.23万
• 依托单位: JOHNS HOPKINS UNIVERSITY
• 依托单位国家: 美国
• 财政年份: 1998
• 资助国家: 美国
• 起止时间: 1998-12-01 至 1999-11-30
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/6218132
• 关键词: butyrates; child (0-11); clinical research; dosage; human subject; human therapy evaluation; inborn urea cycle disorder; metabolism disorder chemotherapy; pediatric pharmacology; phenylcarboxylate

This project is a phase III study of the safety and effectiveness of high dose sodium phenylbutyrate in the management of inborn errors or urea synthesis. Results to the data suggests improved survival and intellectual development.

该项目是对高剂量苯丁酸钠在先天错误或尿素合成管理中的安全性和有效性的研究。 数据的结果表明生存和智力发展改善。