NATURAL HISTORY AND TREATMENT OF GAUCHER DISEASE

本子项目是利用由NIH/NCRR资助的中心赠款提供的资源的众多研究子项目之一。子项目和研究者（PI）可能已经从另一个NIH来源获得了主要资金，因此可以在其他CRISP条目中表示。列出的机构是中心的，不一定是研究者的机构。这是一项评价戈谢病的自然病史和治疗的研究。它将寻求确定一种治疗方案的安全性和有效性，在这种治疗方案中，对有症状的戈谢病患者，初始使用中等剂量的Ceredase（一种市售形式的胎盘酸β -葡萄糖苷酶），然后使用较低的维持剂量。

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.