IRCT20181023041435N1招募中4 期
Survey of Miglustat therapeutic effects on neurological and systemic symptoms of infantile type of Sandhoff and Tay-Sachs diseases
适应症
试验速览
- 阶段
- 4 期
- 状态
- 招募中
- 入组人数
- 30
研究概览
简要总结
暂无简介。
研究设计
- 研究类型
- Interventional
入排标准
- 年龄范围
- 6 months 至 24 months（—）
- 性别
- All
入选标准
- •Clinically and genetically confirmed cases of Sandhoff (SD)/Tay-Sachs (TSD) diseases
- •Enrolled patients who are referred to Myelin Disorders Clinic, Children’s Medical Center with clinically suspicion to TSD and SD
排除标准
- •Renal impairement
- •Loss of follow up
- •Other systemic diseases
- •Concomitant drug therapy which may affect neurological system function
研究者
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