NATURAL HISTORY AND TREATMENT OF GAUCHER DISEASE

该子项目是利用NIH/NCRR资助的中心赠款提供的资源的许多研究子项目之一。子项目和研究者（PI）可能从另一个NIH来源获得主要资金，因此可以在其他CRISP条目中表示。所列机构为中心，不一定是研究者所在机构。这是一项研究性研究，旨在评价戈谢病的自然史和治疗。它将寻求确定一种治疗方案的安全性和有效性，其中在症状性戈谢病患者中，初始中等剂量的Ceredase（一种市售形式的胎盘酸β-葡萄糖苷酶）随后是较低的维持剂量。

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 is a research study to evaluate the natural history and treatment for Gaucher disease. It will seek to determine the safey and efficacy of a treatment regimen in which an initial moderate dose of Ceredase, a commercially available form of placental acid beta-glucosidase is followed by lower maintenance dosing in symptomatic Gaucher disease patients.