EUCTR2005-001629-27-IT进行中（未招募）不适用
A Long-term Continuation Study of Patients With Infantile-Onset Pompe Disease WhoWere Previously Enrolled in Protocol AGLU01602
适应症
试验速览
- 阶段
- 不适用
- 状态
- 进行中（未招募）
- 发起方
- GENZYME
研究概览
简要总结
暂无简介。
研究设计
- 研究类型
- Interventional clinical trial of medicinal product
入排标准
- 性别
- All
入选标准
- •Are the trial subjects under 18? yes
- •Number of subjects for this age range:
- •F.1.2 Adults (18-64 years) no
- •F.1.2.1 Number of subjects for this age range
- •F.1.3 Elderly (>=65 years) no
- •F.1.3.1 Number of subjects for this age range
排除标准
- 未提供
研究者
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A Long-term Continuation Study of Patients with Infantile-Onset Pompe Disease who were previously enrolled in Protocol AGLU01602Pompe disease is a rare metabolic muscle disease inherited in an autosomal recessive fashion. Pompe disease is caused by a deficiency of GAA, which is needed for the degradation of lysosomal glycogen. Pompe disease is characterized by organelle bound (lysosomal) accumulation of glycogen in many body tissues. In general, there is an inverse correlation between the amount of residual GAA activity in patients with Pompe disease and the severity of the disease.EUCTR2005-001629-27-DEGenzyme Europe BV17
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