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药物发现与人类非洲锥虫病:一种较少被忽视的疾病?

Drug discovery and human African trypanosomiasis: a disease less neglected?

机构信息

Medicinal Chemistry, Monash Institute of Pharmaceutical Sciences, Monash University (Parkville Campus), 381 Royal Parade, Parkville, VIC 3052, Australia.

出版信息

Future Med Chem. 2013 Oct;5(15):1801-41. doi: 10.4155/fmc.13.162.

Abstract

Human African trypanosomiasis (HAT) has been neglected for a long time. The most recent drug to treat this disease, eflornithine, was approved by the US FDA in 2000. Current treatments exhibit numerous problematic side effects and are often ineffective against the debilitating CNS resident stage of the disease. Fortunately, several partnerships and initiatives have been formed over the last 20 years in an effort to eradicate HAT, along with a number of other neglected diseases. This has led to an increasing number of foundations and research institutions that are currently working on the development of new drugs for HAT and tools with which to diagnose and treat patients. New biochemical pathways as therapeutic targets are emerging, accompanied by increasing numbers of new antitrypanosomal compound classes. The future looks promising that this collaborative approach will facilitate eagerly awaited breakthroughs in the treatment of HAT.

摘要

人体感染非洲锥虫病(HAT)长期以来一直被忽视。最近一种治疗该病的药物依氟鸟氨酸于 2000 年获得美国食品药品监督管理局(FDA)批准。目前的治疗方法存在许多有问题的副作用,而且往往对疾病使人虚弱的中枢神经系统(CNS)居留期无效。幸运的是,在过去的 20 年中,为了消灭 HAT 和其他一些被忽视的疾病,已经形成了若干伙伴关系和倡议。这导致目前有越来越多的基金会和研究机构致力于开发治疗 HAT 的新药和用于诊断和治疗患者的工具。新的生化途径作为治疗靶点正在出现,同时也出现了越来越多的新型抗锥虫化合物类别。前景看好的是,这种合作方法将促进人们期待已久的在治疗 HAT 方面的突破。

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