Paroxysmal nocturnal haemoglobinuria (PNH) is a progressive and life-threatening disease that causes thrombosis, end organ damage and impaired quality of life. Chronic uncontrolled complement activation leads to chronic haemolysis, causing progressive morbidities and early mortality. Hence, early diagnosis is essential for improved patient management and prognosis. Eculizumab (SOLIRIS®) specifically inhibits chronic, uncontrolled complement activation and is the first-in-class, humanised, monoclonal antibody targeting C5 within the terminal complement pathway. Eculizumab is the first and only approved treatment for PNH in adults and children.

中文翻译：

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从超过25年的PNH经验中学到的东西：靶向补体抑制的时代。

阵发性夜间血红蛋白尿（PNH）是一种进行性且危及生命的疾病，可导致血栓形成，末梢器官损害和生活质量受损。慢性不受控制的补体激活导致慢性溶血，导致进行性发病和早期死亡。因此，早期诊断对于改善患者管理和预后至关重要。依库丽单抗（SOLIRIS®）特别抑制慢性，不受控制的补体激活，并且是末端补体途径中靶向C5的同类首创的人源化单克隆抗体。Eculizumab是成人和儿童中PNH的首个也是唯一获批的治疗方法。