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Translating Peroxisome Biogenesis Disorders: Identifying Pharmacological Therapies and Clinical Trial Endpoints (PERescue)

Translating Peroxisome Biogenesis Disorders: Identifying Pharmacological Therapies and Clinical Trial Endpoints (PERescue)
转化过氧化物酶体生物合成障碍：确定药理学疗法和临床试验终点 (PERescue)
批准号：
332018
负责人：
Braverman Nancy E
金额：
$23.66万
依托单位：
Research Institute of the McGill University Health Centre
依托单位国家：
加拿大
项目类别：
Operating Grants
财政年份：
2015
资助国家：
加拿大
项目状态：
已结题
起止时间：
2015-06-01 至 2018-06-01
来源：
https://webapps.cihr-irsc.gc.ca/decisions/p/project_details.html?applId=332018&lang=en
关键词：
Drug Therapies Model Systems Of Disease Molecular Medicine Natural History Of Disease Patient Registries Peroxisome Biogenesis Disorders Plasmalogen Replacement Therapy

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英文摘要
Peroxisome biogenesis disorders (PBD) are rare autosomal recessive disorders caused by defects in any one of 14 PEX genes, whose protein products are required for peroxisome assembly. Defective peroxisome assembly results in multiple peroxisomal enzyme de
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