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; 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.

该副本是利用众多研究子项目之一 由NIH/NCRR资助的中心赠款提供的资源。子弹和 调查员（PI）可能已经从其他NIH来源获得了主要资金， 因此可以在其他清晰的条目中代表。列出的机构是 对于中心，这不一定是调查员的机构。 这是一项研究，旨在评估Gaucher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功效，在这种治疗方案中，初始剂量的CEREDASE（一种可商用形式的胎盘酸β-葡萄糖苷酶）在有症状的Gaucher病患者中降低主t剂量。