ORAL NICTOTINIC ACID FOR TREATMENT OF LESCH NYHAN PATIENTS

口服烟碱酸治疗 LESCH NYHAN 患者

• 批准号: 6118055
• 负责人: J E SEEGMILLER
• 金额: $ 1.46万
• 依托单位: UNIVERSITY OF CALIFORNIA, SAN DIEGO
• 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/6118055
• 关键词: Lesch Nyhan syndrome; clinical research; human subject; human therapy evaluation; hypoxanthine phosphoribosyltransferase; mental disorder chemotherapy; nicotinate; oral administration; pyrophosphates

Evaluate the possible therapeutic benefit of oral doses of nicotinic acid for reducing the high intracellular content of phosphoribosyl-1- pyrophosphate found in patients with Lesch-Nyhan disease and thereby ameliorate the clinical features of the disease.

评估口服烟酸的可能治疗益处可减少在Lesch-nyhan疾病患者中发现的磷酸蛋白酶基-1-焦磷酸的高细胞内含量，从而改善该疾病的临床特征。