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在经历了十多年的失望后,一家创业公司带头开发出了一类基于一种获得过诺贝尔奖的理念的强效新药。患病初始,行动不便的感觉次数增加。把咖啡洒了,上下楼梯时绊倒,遇上原本很容易就能避开的事故——人都会不时跌倒。但情况会不可避免地恶化。随着患者失去行走或用手进行灵活作业的能力,家族性淀粉多神经病(又称FAP)有可能被误诊数年。大多数患者在首发症状后10至15年内死亡。目前无药可医。这种疾病是由肝内产生的畸形蛋白质引起的,因此有一种治疗方法是肝移植。但是很少有患者能获得肝——而且这种治疗方法只能减缓疾病。
After more than a decade of disappointment, a startup started as a potent new drug based on a concept that earned Nobel Prizes. The initial illness, the number of sensory disturbances increased. Spilled coffee, stumbled up and down the stairs, and encountered accidents that could have been easily avoided - people fell from time to time. But the situation will inevitably deteriorate. Familial amyloid polyneuropathy (also known as FAP) may be misdiagnosed for several years as patients lose their ability to walk or flex their hands. Most patients die within 10 to 15 years of the first symptom. Currently no medicine available. This disease is caused by abnormal protein produced in the liver, so there is a treatment for liver transplantation. However, few patients have access to the liver - and this treatment only slows the disease.