Transthyretin (ATTR) amyloidosis is a systemic disorder particularly affecting the heart and nerves resulting from the misfolding and aggregation of transthyretin (TTR) protein into amyloid fibrils. Cardiac amyloidosis can lead to debilitating heart failure, arrhythmias, and poor survival, with a median life expectancy of 2–6 years following diagnosis (1). Although TTR stabilisers like tafamidis have been demonstrated to slow disease progression, emerging gene-silencing therapies are revolutionising the treatment landscape (2,3) . These therapies target TTR protein production at the genetic level, providing more potent disease modification. The recently completed HELIOS-B trial highlights the efficacy of a gene silencer, vutrisiran, in ATTR amyloidosis and cardiomyopathy, advancing hopes for improved outcomes in this challenging condition (4).
Author Heartbeat Sub-Editor: Dr Khin Kay Kay Kyaw I am currently working as cardiology specialist registrar ST6 at southwest peninsula region while also working as
Author Sub-editor: Dr Pok-Tin Tang Dr Pok-Tin Tang is an ST5 cardiology registrar in the Thames Valley deanery, currently undertaking a period of out of
Author Sub-editor: Dr May Hu Dr May Hu is a cardiology trainee in the North West deanery. She graduated with First Class Honours from the University
Author Sub-editor: Dr Jhiamluka Solano Dr Jhiamluka Solano is a cardiology resident doctor (ST6) in the Yorkshire and Humber Deanery, currently undertaking a DPhil (PhD)