60-Second Journal Club: Vutrisiran in ATTR Amyloidosis with Cardiomyopathy

Transthyretin amyloidosis, also called ATTR amyloidosis, is a progressive, fatal disease caused by misfolded transthyretin (TTR) protein that accumulates as amyloid fibrils in multiple organs, commonly leading to cardiomyopathy.

In the HELIOS-B trial, researchers assessed the efficacy and safety of vutrisiran, an RNA interference therapeutic that inhibits the synthesis of transthyretin, in patients with ATTR amyloidosis with cardiomyopathy. NEJM Editorial Fellow Christos Kotanidis, MD, DPhil, summarizes the results in this 60-Second Journal Club.

Read the full study: https://nej.md/3X5nl5Q

#cardiology #cardiomyopathy #genetherapy #clinicaltrials #nejm