Amyloid transthyretin amyloidosis (ATTR) is a progressive and often fatal disease caused by the buildup of mutated (hereditary ATTR [hATTR]; also known as ATTR variant [ATTRv]) or normal transthyretin (wild‐type ATTR) throughout the body. Two new therapies—inotersen, an antisense oligonucleotide therapy, and patisiran, an RNA interference therapy—received marketing authorization and represent a significant advance in the treatment of amyloidosis. Herein, we describe the clinical presentation of ATTR, commonly used procedures in its diagnosis, and current treatment landscape for ATTR, with a focus on hATTR.

中文翻译：

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遗传性运甲状腺素蛋白淀粉样变性的治疗进展：综述。

淀粉样蛋白甲状腺素蛋白淀粉样变性病（ATTR）是一种进展性疾病，通常是致命的疾病，由突变（遗传性ATTR [hATTR]；也称为ATTR变体[ATTRv]）或正常的甲状腺素蛋白（野生型ATTR）的积累引起。两种新疗法（一种反义寡核苷酸疗法inotersen和一种RNA干扰疗法patisiran）获得了市场许可，代表了淀粉样变性治疗的重大进展。在这里，我们描述ATTR的临床表现，诊断中常用的程序以及ATTR的当前治疗前景，重点是hATTR。