CORRELATION OF MARKERS OF METABOLIC CONTROL WITH LONG-TERM COMPLICATIONS IN GSD

代谢控制标志物与 GSD 长期并发症的相关性

• 批准号: 7950725
• 负责人: David Daniel Weinstein
• 金额: $ 7.52万
• 依托单位: UNIVERSITY OF FLORIDA
• 依托单位国家: 美国
• 财政年份: 2008
• 资助国家: 美国
• 起止时间: 2008-12-01 至 2009-07-31
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/7950725
• 关键词: Age; Anemia; Biochemical; Clinical; Clinical Research; Computer Retrieval of Information on Scientific Projects Database; Development; Diagnosis; Disease; Funding; Generations; Glycogen Storage Disease; Grant; Hepatic; Institution; Kidney; Metabolic Control; Natural History; Patients; Population; Primary carcinoma of the liver cells; Research; Research Personnel; Resources; Role; Source; United States National Institutes of Health; adenoma

This subproject is one of many research subprojects utilizing the resources provided by a Center grant funded by NIH/NCRR. The subproject and investigator (PI) may have received primary funding from another NIH source, and thus could be represented in other CRISP entries. The institution listed is for the Center, which is not necessarily the institution for the investigator. This natural history study is necessary to understand glycogen storage disease as people diagnosed with this disease age. Since the treatment for glycogen storage disease was first implemented approximately 30 years ago, the first generation of patients is now surviving into adulthood. It is important to follow these patients and assess the status of the population. While clinical advances have resulted in amelioration of the biochemical abnormalities in the glycogen storage diseases, long-term complications continue to occur. This study will attempt to understand the role of metabolic control on the development of complications. In particular, the effect of metabolic control on hepatic adenoma formation, hepatocellular carcinoma development, renal complications, and anemia will be assessed.

该子项目是利用该技术的众多研究子项目之一 资源由 NIH/NCRR 资助的中心拨款提供。子项目及 研究者 (PI) 可能已从 NIH 的另一个来源获得主要资金， 因此可以在其他 CRISP 条目中表示。列出的机构是 对于中心来说，它不一定是研究者的机构。 这项自然史研究对于了解糖原贮积症患者的年龄是必要的。 自大约 30 年前首次实施糖原贮积症治疗以来，第一代患者现已存活至成年。 跟踪这些患者并评估人群的状况非常重要。 虽然临床进展已经改善了糖原贮积病的生化异常，但长期并发症仍在继续发生。 这项研究将试图了解代谢控制对并发症发生的作用。 特别是，将评估代谢控制对肝腺瘤形成、肝细胞癌发展、肾脏并发症和贫血的影响。