ISRCTN96480891已完成未知
Cholic acid treatment in Peroxisomal Biogenesis Disorders (Zellweger spectrum): biochemical and clinical effects
Emma Paeditric Hosptial (Netherlands)0 个研究点目标入组 25 人开始时间: 2012年1月30日最近更新:
试验速览
- 阶段
- 未知
- 状态
- 已完成
- 发起方
- 入组人数
- 25
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
- 性别
- All
入选标准
- •1. Zellweger spectrum disorder
- •2. At least one of the following hallmarks:
- •2.1. Steatorrhea
- •2.2. Elevated transaminases
- •2.3. Growth retardation
- •2.4. Neurological symptoms
排除标准
- •Short life expectancy (severe multiple organ dysfunction at the time of diagnosis)
研究者
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