National Registry for Egyptian Pediatric Neuromuscular Diseases
试验速览
- 阶段
- 不适用
- 入组人数
- 200
- 试验地点
- 1
- 主要终点
- Functional motor ability
研究概览
简要总结
Our aim is to establish multi-center national Egyptian database of information for inherited and acquired neuromuscular diseases in infants and children from 0 to 18 years of age.
详细描述
Aims: An open-ended multi-center, national Egyptian study to collect and analyze data for children with Neuromuscular Diseases (NMD) inherited NMD (spinal muscular atrophy (SMA), Duchenne/Becker and congenital muscular dystrophies (DMD/BMD, CMD), congenital myopathies, and congenital myasthenic syndromes) and acquired NMD (neuropathies, myasthenia gravis and myositis).
Participants: Eligible infants and children with inherited and acquired neuromuscular diseases.
DESIGN: This study is a prospective cohort study.
Outcome measures: Motor development assessment, respiratory and cardiac examination.
研究设计
- 研究类型
- Observational
- 观察模型
- Case Only
- 时间视角
- Prospective
入排标准
- 年龄范围
- 1 Month 至 18 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Weakness, hypotonia.
- •Nerve conduction study and electromyographic confirmation of lower motor neuron affection.
排除标准
- •Chromosomal diseases.
- •Malformations and deformations.
结局指标
主要结局
Functional motor ability
时间窗: 1 year
Motor power in acquired acute neuromuscular diseases will be assessed at admission and morbidity and mortality at discharge from hospital. Functional motor ability will be performed every 3 months for children with inherited neuromuscular diseases.
次要结局
- Cardio-pulmonary function(12 months)
研究者
Sahar M.A. Hassanein, MD
Professor of Pediatrics
Ain Shams University