NATURAL HISTORY AND TREATMENT OF GAUCHER DISEASE

戈谢病的自然史和治疗

• 批准号: 7953651
• 负责人: MANISHA BALWANI
• 金额: $ 33.13万
• 依托单位: ICAHN SCHOOL OF MEDICINE AT MOUNT SINAI
• 依托单位国家: 美国
• 财政年份: 2009
• 资助国家: 美国
• 起止时间: 2009-03-01 至 2009-07-31
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/7953651
• 关键词: Clinical Research; 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.

这个子项目是许多利用 由NIH/NCRR资助的中心赠款提供的资源。子项目和 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。 这是一项研究性研究，旨在评价戈谢病的自然史和治疗。 本研究将试图确定一种治疗方案的安全性和有效性，在该方案中，有症状的戈谢病患者初始中等剂量的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。