Abstract：
Transthyretin amyloid cardiomyopathy (ATTR-CM) is caused by the myocardial extracellular deposition of amyloid fibrils formed from the dissociation of TTR tetramer into monomers. The rate-limiting step in TTR amyloidogenesis is the dissociation of the TTR tetramer into monomers: Tafamidis is an effective TTR-stabilizer in its native homotetrameric structure. Tafamidis is a safe and effective drug in reducing symptoms, hospitalization and mortality in accurately selected patients affected by hereditary and wild-type transthyretin amyloid cardiomyopathy.
摘要：
转甲状腺素蛋白淀粉样心肌病（ATTR-CM）是由TTR四聚体解离成单体而形成的淀粉样原纤维的心肌细胞外沉积引起的。TTR淀粉样生成中的限速步骤是将TTR四聚体解离成单体：Tafamidis在其天然同源四聚体结构中是有效的TTR稳定剂。Tafamidis是一种安全有效的减轻症状的药物，准确选择的遗传性和野生型甲状腺素运载蛋白淀粉样心肌病患者的住院率和死亡率。
