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Precision Health for Neuromuscular Diseases: a bench-to-bedside-to-bench approach to discover their genetic cause, unravel their molecular pathways, identify biomarkers, and conduct clinical trials for patient benefit.

Precision Health for Neuromuscular Diseases: a bench-to-bedside-to-bench approach to discover their genetic cause, unravel their molecular pathways, identify biomarkers, and conduct clinical trials for patient benefit.
神经肌肉疾病的精准健康：通过从实验室到床边到实验室的方法来发现其遗传原因，揭示其分子途径，识别生物标志物并进行临床试验以造福患者。
批准号：
390200
负责人：
Lochmüller Hanns
金额：
$340.66万
依托单位：
Children's Hospital of Eastern Ontario- Ottawa Children's Treatment Centre
依托单位国家：
加拿大
项目类别：
Operating Grants
财政年份：
2018
资助国家：
加拿大
项目状态：
未结题
起止时间：
2018-09-01 至 2025-09-01
来源：
https://webapps.cihr-irsc.gc.ca/decisions/p/project_details.html?applId=390200&lang=en
关键词：
Clinical Biomarkers Clinical Trials Deep Phenotyping Drug Repurposing Gene Discovery Genomics Molecular Pathways Neuromuscular Disease Rare Disease Translational Research

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英文摘要
Voluntary movement of muscles in humans requires electric signalling down the nerves: beginning at the spinal cord and ending at muscles that help us speak, eat, breathe and move. This process is impaired in neuromuscular diseases (NMDs), which are associ
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