A STUDY OF THE DOSING AND EFFICACY OF MODIFIED RESISTANT CORNSTARCH IN GSD 1A PA

这个子项目是许多研究子项目中的一个 由NIH/NCRR资助的中心赠款提供的资源。子项目及 研究者（PI）可能从另一个NIH来源获得了主要资金， 因此可以在其他CRISP条目中表示。所列机构为 研究中心，而研究中心不一定是研究者所在的机构。 糖原累积病是一组遗传性疾病，其中表型是严重的低血糖症，因为不能从肝脏和肾脏中的糖原产生葡萄糖。 I型糖原累积病GSD 1是由肝葡萄糖-6-磷酸酶缺乏引起的，肝葡萄糖-6-磷酸酶是糖原分解和糖原生成的组成部分。 缺乏这种酶会导致无法维持正常的葡萄糖稳态和严重的空腹低血糖。 目前，患者平均每天用6.2剂玉米淀粉治疗。 需要一种可消化的淀粉，其可以在GSD患者的餐后提供更长时间的葡萄糖来源。 维生素淀粉是一种高淀粉酶，热改性的蜡质玉米淀粉，最近被发现与传统的玉米淀粉相比，在糖原累积病1a和1b型中具有较低的血糖指数和更长的持续时间。 现在提出了一项研究，以确定这种新产品的最佳剂量，并确定新产品的量的修改是否会提供生化益处和改善GSD 1a患者的葡萄糖浓度维持。

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. The glycogen storage diseases GSD are a set of genetic disorders in which the phenotype is severe hypoglycemia because of the inability to produce glucose from glycogen in the liver and kidney. Type I glycogen storage disease GSD1 is caused by a deficiency of hepatic glucose-6-phosphatase, which is an integral part of both glycogenolysis and gluconeogenesis. Lack of this enzyme results in the inability to maintain normal glucose homeostasis and severe fasting hypoglycemia. Patients presently are treated with an average of 6.2 doses of cornstarch per day. There is a need for a digestible starch that can provide a source of glucose for longer duration after meals in GSD patients. Vitastarch is a high amylase, heat modified waxy cornstarch which recently was found to have a lower glycemic index and be longer lasting when compared with traditional cornstarch in glycogen storage disease types 1a and 1b. A study is now proposed to determine the optimal dosing of this new product and to determine if modification of the amount of the new product will offer biochemical benefit and improved maintenance of glucose concentrations in patients with GSD 1a.