Zhang W, Zhao X Y, Huang J, Ou X J, Jia J D
Liver Research Center, Beijing Friendship Hospital, Capital Medical University, Beijing 100050, China National Clinical Research Center for Digestive Diseases, Beijing 100050, China.
National Clinical Research Center for Digestive Diseases, Beijing 100050, China Beijing Institute of Clinical Medicine, Beijing Friendship Hospital, Capital Medical University, Beijing 100050, China.
Zhonghua Gan Zang Bing Za Zhi. 2024 Sep 20;32(9):783-786. doi: 10.3760/cma.j.cn501113-20240714-00324.
Wilson's disease, also known as hepatolenticular degeneration, is an inherited disorder of copper metabolism caused by homozygous or compound heterozygous variants in the 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.
威尔逊病，又称肝豆状核变性，是一种由该基因的纯合或复合杂合变异引起的铜代谢遗传性疾病，主要临床表现为肝脏疾病和/或神经/心理障碍，以及周边角膜的凯-弗环。目前，威尔逊病患者需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，但仍存在未满足的临床需求，因为一些接受治疗的患者疗效不佳、疾病进展或出现严重药物不良反应。近年来，新型治疗药物发展迅速。本文将总结威尔逊病药物治疗的进展，为威尔逊病的治疗提供新的思路。
