CEREDASE TREATMENT OF GAUCHER DISEASE

戈谢病的 CEREDASE 治疗

• 批准号: 3849017
• 负责人: GREGORY A GRABOWSKI
• 金额: --
• 依托单位: MOUNT SINAI SCHOOL OF MEDICINE OF CUNY
• 依托单位国家: 美国
• 资助国家: 美国
• 起止时间: 至
• 项目状态: 未结题
• 来源: https://reporter.nih.gov/project-details/3849017
• 关键词: Gaucher's disease; child (0-11); enzyme therapy; glucosylceramidase; human subject; human therapy evaluation; molecular pathology

Objective of the research is to evaluate the efficacy of enzyme replacement for the alleviation of signs and symptoms of Gaucher disease Type 1. The goal is to develop techniques for early assessment of therapeutic effectiveness. Long term objective is to determine prospectively the relationship of disease severity, molecular defect and response to therapy.

本研究的目的是评价酶的功效 缓解戈谢病体征和症状的替代品 1型。 目标是开发早期评估的技术， 治疗效果长期目标是确定 前瞻性地研究了疾病严重程度、分子缺陷和 对治疗的反应。