In this edition of ESC Bench to Bedside, we focus on the evolving management of transthyretin amyloidosis cardiomyopathy (ATTR-CM). The issue opens with a review of disease biology and the mechanisms of action for stabilisers, silencers, antibody therapies, and gene editing, highlighting how each approach targets distinct steps in the amyloid cascade.
We then critically appraise the latest clinical trial data – including results from ATTR-ACT, APOLLO-B, HELIOS-B, CARDIO-TTRansform, ATTRibute-CM, NI006 / ALXN2220, PRX004, and NTLA-2001 – to support informed comparisons of efficacy and safety. Finally, we offer evidence-based recommendations for treatment sequencing and patient selection, supporting clinicians in the practical application of these advances.
