NATURAL HISTORY AND TREATMENT OF GAUCHER DISEASE

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。 这是一项研究，旨在评估高谢病的自然病史和治疗方法。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，对有症状的高谢病患者首先服用中等剂量的Ceredase，这是一种商业形式的胎盘酸β-葡萄糖苷酶，然后再给予较低的维持剂量。

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 treat-ment regimen in which an initial moderate dose of Ceredase, a commercially available form of placental acid beta-glucosidase is followed by lower main-tenance dosing in symptomatic Gaucher disease patients.