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继三次拒绝之后,欧洲药品监管局已建议批准西方国家第一个基因治疗药物Glybera,这在创新药领域中是一个重要的突破。自从应用创新方法用于修复基因缺陷的首次试验开始20多年来,科学家和制药公司一直致力于基因治疗的实践中。Glybera是极罕见遗传病脂蛋白脂酶缺乏(LPLD)患者的一个可能的救命索。LPLD患者不能处理血浆中的脂肪颗粒,无法进行正常饮食。据估计,每一百万人中不超过一、两个人患有此种疾病,这种疾病可以引起急性胰腺炎,从而导致死亡。
Following three refusals, the European Medicines Agency has approved the approval of Glybera, the country’s first gene therapy drug, which is an important breakthrough in the field of innovative medicines. More than two decades have passed since the first trial of applying innovative approaches to repair genetic defects, scientists and pharmaceutical companies have been dedicated to the practice of gene therapy. Glybera is a potential lifeline for patients with extremely rare inherited lipoprotein lipase deficiency (LPLD). LPLD patients can not handle the fat particles in the plasma, can not be normal diet. It is estimated that no more than one in two million people suffer from the disease, which can cause acute pancreatitis, leading to death.