CTRI/2018/03/012267尚未招募未知
Genzyme Rare disease registries (Pompe Disease Registry Protocol)
Sanofi Synthelabo India Private Limited0 个研究点目标入组 0 人开始时间: 待定最近更新:
试验速览
- 阶段
- 未知
- 状态
- 尚未招募
- 发起方
研究概览
简要总结
暂无简介。
研究设计
- 研究类型
- Observational
入排标准
入选标准
- •1.All age group from new born to old age allowed.
- •2. All patients with a confirmed diagnosis of Pompe disease who are willing and able to provide written informed consent and any additional authorization documents required by local law to send health information to the Registry are eligible for inclusion, regardless of whether they are receiving disease therapy including ERT (such as alglucosidase alfa) and irrespective of the commercial product with which they are being treated.
- •3. A confirmed diagnosis is defined as documented acid α-glucosidase (GAA) enzyme deficiency from blood, skin, or muscle tissue and/or documentation of 2 GAA gene mutations.
排除标准
- •No Exclusion Criteria
研究者
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