治疗转甲状腺素蛋白介导的淀粉样变性心肌病新药：氯苯唑酸

转甲状腺素蛋白(TTR)介导的淀粉样变性心肌病主要是由野生或突变型TTR淀粉样纤维错误折叠在心肌沉积所致。氯苯唑酸对TTR的甲状腺素结合部位具有较高的亲和力,可选择性与其结合并抑制TTR降解。临床试验表明,氯苯唑酸可使90%以上患者的TTR稳定,而且与安慰剂比较,氯苯唑酸可明显降低患者的全因死亡率及心血管疾病相关住院率,患者心脏功能和生活质量也显著改善。氯苯唑酸无严重不良事件,患者耐受性好。

A new drug for treatment of transthyretin amyloid cardiomyopathy: tafamidis

Transthyretin(TTR)amyloid cardiomyopathy is caused by the deposition of TTR amyloid fibrils in the myocardium.The deposition occurs when wild-type or variant TTR becomes misfolds.Tafamidis binds to the thyroxinebinding sites of TTR with high affinity and selectivity and inhibits the dissociation of tetramers into monomers.Clinical trials indicated that TTR stabilization was achieved in more than 90% of patients who treated with tafamidis,and tafamidis was associated with reductions in all-cause mortality and cardiovascular-related hospitalizations and reduced the decline in functional capacity and quality of life as compared with placebo.There were no serious adverse events in administration of tafamidis and patients were well tolerated.