The full scope of its physiologic role and mechanism of degradation remain incompletely elucidated however, the predominant hypothesis is that the protein is naturally unstable and prone to misfolding in-vivo. Transthyretin (TTR), also known as prealbumin, is a protein synthesized primarily by the liver and is responsible for the transportation of thyroxine hormone and retinol-binding protein. Transthyretin amyloid cardiomyopathy (ATTR-CM) is restrictive cardiomyopathy caused by an abnormal extracellular deposition of amyloid fibrils in the myocardium. ![]() This review article provides a survey of the pharmacokinetic and clinical data on all currently available treatments. Treatments broadly fall into three categories: (1) TTR silencing through mRNA knockdown or silencing (2) TTR stabilization and (3) TTR resorption or extraction. Several advances in pharmacotherapeutic treatments have significantly reduced the morbidity and mortality of the disease. Recognition of ATTR-CM has been aided by rapid advances in technologies to diagnose the disease more accurately. The TTR protein itself can be either wild-type (ATTRwt) or one of many pathologic variants (ATTRv). ATTR-CM is caused by an aggregation of misfolded transthyretin (TTR) protein amyloid fibrils in the myocardium. Transthyretin amyloidosis of the heart, or transthyretin amyloid cardiomyopathy (ATTR-CM), once thought to be a rare disease, is now increasingly recognized as a common causing of restrictive cardiomyopathy, particularly in elderly patients and patients with heart failure with preserved ejection fraction.
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