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

这个子项目是许多研究子项目中利用 资源由NIH/NCRR资助的中心拨款提供。子项目和 调查员(PI)可能从NIH的另一个来源获得了主要资金， 并因此可以在其他清晰的条目中表示。列出的机构是 该中心不一定是调查人员的机构。 这项自然历史研究对于了解糖原储存疾病是必要的，因为人们被诊断为这种疾病的年龄。自从大约30年前首次实施糖原储存疾病的治疗以来，第一代患者现在可以存活到成年。重要的是要跟踪这些患者并评估人群的状况。虽然临床进展导致糖原储存疾病的生化异常得到改善，但长期并发症仍在继续发生。本研究试图了解代谢控制在并发症发生中的作用。特别是，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将被评估。

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, hepatocelluar carcinoma development, renal complications, and anemia will be assessed.