Treatment of Transthyretin Amyloid Cardiomyopathy: The Current Options, the Future, and the Challenges.

ABSTRACT: Transthyretin amyloid cardiomyopathy (ATTR-CM) is a progressively debilitating, rare disease associated with high mortality. ATTR-CM occurs when TTR amyloid protein builds up in the myocardium along with different organs, most commonly the peripheral and the autonomic nervous systems. Managing the cardiac complications with standard heart failure medications is difficult due to the challenge to maintain a balance between the high filling pressure associated with restricted ventricular volume and the low cardiac output. To date, tafamidis is the only agent approved for ATTR-CM treatment. Besides, several agents, including green tea, tolcapone, and diflunisal, are used off-label in ATTR-CM patients. Novel therapies using RNA interference also offer clinical promise. Patisiran and inotersen a

SUBMITTER: Tschope C 

PROVIDER: S-EPMC9031576 | biostudies-literature | 2022 Apr

REPOSITORIES: biostudies-literature