NL-OMON39837已完成2 期
Cholic acid treatment in Peroxisomal Biogenesis Disorders (Zellweger spectrum): biochemical and clinical effects. - Cholic acid in Zellweger
适应症
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 入组人数
- 20
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
- 2 至 99（—）
入选标准
- •Phenotypically mild Zellweger spectrum disorder.
- •At least one of the following hallmarks: steatorrhea, elevated transaminases, growth retardation, neurological symptoms
排除标准
- •Short life expectancy based on severe multiple organ dysfunction at the time of diagnosis
研究者
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