关键词: Alport综合征, 遗传性肾病, 肾素-血管紧张素-醛固酮系统抑制剂, 临床试验, 新药研发

Abstract: Alport syndrome (AS) is a kind of hereditary collagen disease with kidney, eye and hearing impairment as primary manifestations, which will lead to end-stage kidney disease (ESKD)in the early stage, and is included in the first batch of rare diseases list in China. At present, there is no cure for AS, but the research and development of drugs for the treatment of AS is accelerating. This article reviews the progress of drug research and clinical trials of AS at home and abroad in the past decade, hoping to provide strategies and ideas for the future drug development and treatment of AS, so as to benefit AS patients and their families.

Key words: Alport syndrome, hereditary nephropathy, renin-angiotensin-aldosterone system inhibitors, clinical trialnew drug development