NATURAL HISTORY AND TREATMENT OF GAUCHER DISEASE

戈谢病的自然史和治疗

• 批准号: 7718102
• 负责人: MANISHA BALWANI
• 金额: $ 80.93万
• 依托单位: ICAHN SCHOOL OF MEDICINE AT MOUNT SINAI
• 依托单位国家: 美国
• 财政年份: 2008
• 资助国家: 美国
• 起止时间: 2008-03-01 至 2009-02-28
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/7718102
• 关键词: Computer Retrieval of Information on Scientific Projects Database; Dose; Funding; Gaucher Disease; Grant; Institution; Natural History; Patients; Research; Research Personnel; Resources; Source; Treatment Protocols; United States National Institutes of Health; ceredase; glucosylceramidase; research study

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.

该子项目是利用该技术的众多研究子项目之一 资源由 NIH/NCRR 资助的中心拨款提供。子项目和 研究者 (PI) 可能已从 NIH 的另一个来源获得主要资金， 因此可以在其他 CRISP 条目中表示。列出的机构是 对于中心来说，它不一定是研究者的机构。 这是一项评估戈谢病自然史和治疗的研究。 它将寻求确定一种治疗方案的安全性和有效性，其中对有症状的戈谢病患者使用初始中等剂量的 Ceredase（一种市售形式的胎盘酸性 β-葡萄糖苷酶），然后采用较低的维持剂量。