Thelansis

  Transthyretin amyloid cardiomyopathy (ATTR-CM) is a form of systemic amyloidosis characterized by misfolded transthyretin (TTR) protein deposition in the myocardium. Another notable cause of cardiac amyloidosis is the accumulation of immunoglobulin light-chain (AL) aggregates. Although several amyloidogenic proteins may be deposited in various organs and tissues, myocardial involvement is rare. Symptoms of ATTR-CM can vary, and the condition is frequently misdiagnosed, with early stages resembling symptoms of other conditions such as hypertensive heart failure or hypertrophic cardiomyopathy. Some patients may be asymptomatic, while others may progress to end-stage heart failure. The disease is classified based on the sequence of the TTR gene, distinguishing between wtATTR-CM (no mutation) and hATTR-CM (presence of a mutation). In 2019, the FDA approved tafamidis and tafamidis meglumine for treating ATTR-CM. Both agents selectively bind to the transthyretin tetramer, preventing ...