[Progress in drug therapy of Wilson's disease].

W Zhang, X Y Zhao, J Huang, X J Ou, J D Jia
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Abstract

Wilson's disease, also known as hepatolenticular degeneration, is an inherited disorder of copper metabolism caused by homozygous or compound heterozygous variants in the ATP7B gene, which is mainly clinically manifested as liver disease and/or neurological/psychological disorders, and Kayser-Fleischer ring in the peripheral cornea. Patients with Wilson's disease are currently treated with lifelong use of chelating agents that promote copper ion excretion and/or zinc agents that reduce copper absorption, but there is still an unmet clinical need because some patients who receive treatment have poor efficacy, disease progression, or serious adverse drug reactions. In recent years, new therapeutic drugs have been developed rapidly. This article will summarize the advances in drug treatment of Wilson's disease, shedding new light on the treatment of Wilson's disease.

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[威尔逊氏病的药物治疗进展]。
威尔逊氏病又称肝细胞变性,是一种遗传性铜代谢紊乱疾病,由 ATP7B 基因的同卵或复合杂合子变异引起,临床上主要表现为肝脏疾病和/或神经/心理障碍,以及外周角膜的 Kayser-Fleischer 环。目前,威尔逊氏病患者可终身使用促进铜离子排泄的螯合剂和/或减少铜吸收的锌剂进行治疗,但由于部分患者接受治疗后疗效不佳、病情恶化或出现严重的药物不良反应,临床需求仍未得到满足。近年来,新的治疗药物得到了快速发展。本文将总结威尔逊氏病药物治疗的进展,为威尔逊氏病的治疗提供新的思路。
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来源期刊
中华肝脏病杂志
中华肝脏病杂志 Medicine-Medicine (all)
CiteScore
1.20
自引率
0.00%
发文量
7574
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[A case of giant hilar cholangiocarcinoma accompanied by hyperbilirubinemia achieved disease stabilization via hepatic artery infusion chemotherapy combined with targeted therapy]. [A mechanistic study of radiotherapy on intratumoral NK cell infiltration augmentation by regulating the EZH2/CXCL10 pathway in hepatocellular carcinoma cells]. [A nomogram prediction model for individualized prediction of the risk of covert (minimal) hepatic encephalopathy occurrence in patients with liver cirrhosis]. [Analysis of the etiology and clinical indicators of infantile cholestasis]. [Application of zinc agents in Wilson's disease].
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