Alport综合征的药物研发进展  

Progress in drug development of Alport syndrome

作　　者：伍琳琳 刘栋 董宁宁(综述) 夏宏光(审校) WU Linlin;LIU Dong;DONG Ningning;XIA Hongguang(Hangzhou PhecdaMed Co.Ltd,Hangzhou 311121,China)

机构地区：[1]杭州天玑济世生物科技有限公司,杭州311121

出　　处：《肾脏病与透析肾移植杂志》2023年第3期270-275,共6页Chinese Journal of Nephrology,Dialysis & Transplantation

摘　　要：Alport综合征(AS)是一种以肾脏、眼部及听力受损为主要表现的遗传性胶原病,可导致患者早期进入终末期肾病(ESKD),已纳入我国《第一批罕见病目录》。目前,AS尚无根治手段,但是治疗AS的药物研发在加速进展。本文综述了近十年来AS的国内外药物研发和临床试验进展,希望为AS的药物研发及治疗提供策略和思路,使AS患者及其家庭获益。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.

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 

分 类 号：R596[医药卫生—内科学]