Alexion制药公司推出多发性夜间血红蛋白尿的有效药物

 
最近开发了一种方法治疗多发性夜间血红蛋白尿(PNH)，这是一种罕见的致残和危及生命的血液疾病，特征为慢性红细胞破坏或者溶血。

溶血会导致PHN患者出现下列1种或者多种症状:严重贫血，嫉妒乏力，周期性疼痛，呼吸困难，肺源性高血压，间歇性黑尿(血红蛋白尿)，肾脏疾病，生

命质量下降，血液凝集(血栓)。PNH经常危及患者生命，据估计诊断后PNH患者中位数生命为10～15年。

Alexion制药公司(Cheshire, CN, USA)获得了美国食品药品管理局 (FDA)批准使用Soliris (eculizumab)治疗PNH患者以降低溶血。PNH患者缺乏一种特异性蛋白，

该蛋白通常保护红细胞免于补体免疫系统的破坏。Soliris是一种补体抑制剂，能够选择性阻断补体。

Safe Effective Therapy Treats Paroxysmal Nocturnal Hemoglobinuria
 
A therapy has been developed for paroxysmal nocturnal hemoglobinuria (PNH), a rare, disabling and life-threatening blood disorder defined by chronic red blood cell

destruction, or hemolysis.

Hemolysis can cause one or more of the following symptoms in patients with PNH: severe anemia, disabling fatigue, recurrent pain, shortness of breath, pulmonary

hypertension, intermittent episodes of dark colored urine (hemoglobinuria), kidney disease, impaired quality of life, and blood clots (thromboses). PNH often strikes people in

the prime of their lives, the estimated median survival for PNH patients is between 10 and 15 years from the time of diagnosis.

Alexion Pharmaceuticals, Inc., (Cheshire, CN, USA) has received marketing approval from the U.S. Food and Drug Administration (FDA) for Soliris (eculizumab) for treatment

of patients with PNH to reduce hemolysis. Patients with PNH are missing a specific protein that normally protects red blood cells from destruction by a component of the

immune system called terminal complement. Soliris, a complement inhibitor prevents hemolysis by selectively blocking terminal complement.