NATURAL HISTORY AND TREATMENT OF GAUCHER DISEASE

戈谢病的自然史和治疗

• 批准号: 7380501
• 负责人: MELISSA P WASSERSTEIN
• 金额: $ 86.11万
• 依托单位: ICAHN SCHOOL OF MEDICINE AT MOUNT SINAI
• 依托单位国家: 美国
• 财政年份: 2006
• 资助国家: 美国
• 起止时间: 2006-04-17 至 2007-02-28
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/7380501
• 关键词: glucosylceramidase

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.

该子项目是利用NIH/NCRR资助的中心赠款提供的资源的许多研究子项目之一。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。列出的机构适用于该中心，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SAFEY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病患者中降低维持剂量。