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Novel therapeutic approaches to target GNE Myopathy

Novel therapeutic approaches to target GNE Myopathy
针对 GNE 肌病的新治疗方法
批准号：
430847
负责人：
Lochmüller Hanns
金额：
$28.31万
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：
2020
资助国家：
加拿大
项目状态：
已结题
起止时间：
2020-06-01 至 2023-06-01
来源：
https://webapps.cihr-irsc.gc.ca/decisions/p/project_details.html?applId=430847&lang=en
关键词：
Biomarkers Glycobiology Gne Myopathy Innovative Therapeutic Strategies Multi-Omic Prodrug Sialic Acid Pathway

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中文摘要
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英文摘要
GNE myopathy (GNEM) is a rare severely debilitating muscle disease which affects 1 to 9:1,000,000 people among all ethnicities, with elevated carrier rates in populations of Eastern Europe and Asian heritage, including Jewish, Persian, East Indian, Arab a
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会议论文
NMD4C: the neuromuscular network for Canada
NMD4C: An integrated research network for patients, scientists, and clinicians to improve outcomes and access to therapies for patients with neuromuscular disorders in Canada.
Inherited disorders of neuromuscular transmission in humans: understanding and manipulating molecular and cellular structure-function relationships at the motoneuron to muscle interface, the neuromuscular synapse, through a translational medicine approach
Canada Research Chair Tier 1
  • 批准号：
    392579
  • 项目类别：
  • 资助金额：
    $101.99万
  • 财政年份：
    2018
  • 负责人：
    Lochmüller Hanns
  • 依托单位：
    University of Ottawa
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