NTBC THERAPY OF TYROSINEMIA TYPE I

I 型酪氨酸血症的 NTBC 治疗

• 批准号: 6244584
• 负责人: JESS G THOENE
• 金额: $ 2.22万
• 依托单位: UNIVERSITY OF MICHIGAN AT ANN ARBOR
• 依托单位国家: 美国
• 财政年份: 1997
• 资助国家: 美国
• 起止时间: 1997-02-20 至 1997-11-30
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/6244584
• 关键词: alpha fetoprotein; aminoacid metabolism; clinical research; clinical trials; drug screening /evaluation; enzyme inhibitors; human subject; human therapy evaluation; metabolism disorder chemotherapy; oxygenases; thiocyanates; tyrosinemias

Tyrosinemia Type I is a lethal inherited inborn error of metabolism which produces cirrhosis of the liver and hepatocellular carcinoma. Until recently, the only effective therapy was liver transplant. The drug NTBC has been developed as an inhibitor of 4-hydroxyphenylpyruvate dioxygenase. The drug is well tolerated and produces dramatic biochemical improvement. Long-term studies of the drug to determine if it avoids cirrhosis and hepatocellular carcinoma are in process and the drug has been adopted by a pharmaceutical company. One patient is being studied at the University of Michigan.

I型酪氨酸血症是一种致命的遗传代谢的遗传遗传误差 产生肝脏和肝细胞癌的肝硬化。 直到最近，唯一有效的疗法是肝移植。 这 药物NTBC已开发为4-羟基苯基丙酮酸的抑制剂 二氧酶。 该药物的耐受性良好并产生戏剧性 生化改进。 对药物的长期研究以确定是否 它避免了肝硬化和肝细胞癌正在进行中，并且 药物已被制药公司采用。 一个病人正在 在密歇根大学学习。