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Novel strategies and therapeutic options for the management of primary biliary cholangitis

机译:治疗原发性胆源性胆管炎的新策略和治疗选择

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Primary biliary cholangitis (PBC) is a chronic autoimmune liver disease. It has a varied course of progression ranging from being completely asymptomatic to aggressive disease leading to cirrhosis and resulting in liver transplantation. In addition, symptoms can be debilitating and can have a major impact on quality of life. For decades, there was only one anti-cholestatic agent available to target this disease and that was only effective in around half of patients, with little or no effect on symptoms. With increasing understanding of the pathogenic mechanisms of PBC and potential targets for drug treatment, pharmaceutical companies have shown a greater interest in this rare disease. A large number of novel therapeutic molecules have been developed and are currently being evaluated. In this review article all the novel molecules in use and in trials targeting cholestasis and symptoms in PBC are discussed.
机译:原发性胆源性胆管炎(PBC)是一种慢性自身免疫性肝病。从完全无症状到导致肝硬化并导致肝移植的侵袭性疾病,其发展过程各不相同。此外,症状可能使人衰弱,并对生活质量产生重大影响。几十年来,只有一种抗胆汁淤积药可针对这种疾病,并且仅对大约一半的患者有效,对症状几乎没有影响。随着对PBC致病机理和药物治疗潜在靶标的了解越来越多,制药公司对这种罕见疾病表现出了更大的兴趣。已经开发了许多新颖的治疗分子,目前正在对其进行评估。在这篇综述文章中,讨论了针对PBC中的胆汁淤积和症状的所有使用和试验中的新型分子。

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