Introduction
Transthyretin amyloid cardiomyopathy (ATTR-CM) is an increasingly recognized form of cardiac amyloidosis characterized by the deposition of misfolded transthyretin (TTR) protein fibrils within the myocardial tissue. Recent therapeutic advances have brought transformative treatment options, including TTR stabilizers, gene-silencing therapies, and emerging genome-editing and amyloid-depleting strategies. Each of these therapies targets distinct biological mechanisms responsible for disease progression. However, the traditional approach to defining and monitoring disease progression predominantly relies on downstream clinical symptoms, biomarker levels, and functional assessments that do not directly reflect the underlying biological activity targeted by these treatments.
