NL-OMON52746已完成不适用
Ex vivo genetic correction of LAMA2 mutations in myogenic stem cells of patients with merosin-deficient congenital muscle dystrophy type 1a (MDC1a) - Genetic correction LAMA2
适应症
试验速览
- 阶段
- 不适用
- 状态
- 已完成
- 入组人数
- 10
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
- 年龄范围
- 18 至 64（—）
入选标准
- •LAMA2 mutation carriers:
- •- Age >18 years
- •- Heterozygous or homozygous LAMA2 c.5562+5G>C mutation
- •- Written informed consent
- •- Written informed consent
- •- Age >18 years
- •- No muscular dystrophy or other disease known to affect muscle morphology or
排除标准
- •MDC1 patients and controls:
- •- No informed consent
- •- Use of anti-coagulants, anti-thrombotics and other medication influencing
- •coagulation
- •- Have a weekly alcohol intake of >= 35 units (men) or >= 24 units (women)
- •- Current history of drug abuse
- •- A history of strokes
- •- Significant concurrent illness
- •- Ongoing participation in other clinical trials
- •- Major surgery within 4 weeks of the visit
- •- Pregnant or lactating women
- •- Patients unable and/or unwilling to comply with treatment and study
- •instructions
- •- Any other factor that in the opinion of the investigator excludes the patient
- •from the study
研究者
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