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隐孢子虫病药物研发:机遇与挑战

Cryptosporidiosis Drug Discovery: Opportunities and Challenges.

作者信息

Manjunatha Ujjini H, Chao Alexander T, Leong F Joel, Diagana Thierry T

机构信息

Novartis Institute for Tropical Diseases, 10 Biopolis Road, #05-01, Singapore 138670.

出版信息

ACS Infect Dis. 2016 Aug 12;2(8):530-7. doi: 10.1021/acsinfecdis.6b00094. Epub 2016 Jul 22.

Abstract

The apicomplexan parasite Cryptosporidium is the second most important diarrheal pathogen causing life-threatening diarrhea in children, which is also associated with long-term growth faltering and cognitive deficiency. Cryptosporidiosis is a parasitic disease of public health concern caused by Cryptosporidium parvum and Cryptosporidium hominis. Currently, nitazoxanide is the only approved treatment for cryptosporidium infections. Unfortunately, it has limited efficacy in the most vulnerable patients, thus there is an urgent need for a safe and efficacious cryptosporidiosis drug. In this work, we present our current perspectives on the target product profile for novel cryptosporidiosis therapies and the perceived challenges and possible mitigation plans at different stages in the cryptosporidiosis drug discovery process.

摘要

顶复门寄生虫隐孢子虫是导致儿童危及生命腹泻的第二重要腹泻病原体,还与长期生长发育迟缓及认知缺陷有关。隐孢子虫病是由微小隐孢子虫和人隐孢子虫引起的一种引起公共卫生关注的寄生虫病。目前,硝唑尼特是唯一被批准用于治疗隐孢子虫感染的药物。不幸的是,它对最脆弱的患者疗效有限,因此迫切需要一种安全有效的隐孢子虫病治疗药物。在这项工作中,我们阐述了我们对新型隐孢子虫病治疗方法的目标产品概况以及在隐孢子虫病药物研发过程不同阶段所面临的已知挑战和可能的应对计划的当前观点。

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