Improvement of Cardiac Structure And Function In Hereditary Transthyretin Amyloidosis Cardiomyopathy With Inotersen: A Case Report

Hereditary transthyretin amyloidosis (hATTR) is a progressive, fatal disease caused by systemic deposition of misfolded transthyretin (TTR) fibrils resulting in cardiomyopathy (CM) and polyneuropathy (PN). Treatment options include stabilizers (tafamidis) to prevent TTR tetramer dissociation, silencers (inotersen, patisiran) that inhibit hepatic TTR synthesis, or liver transplantation. Silencers have favorable outcomes in PN, with limited data on CM. This case highlights improvement in hATTR-CM in a patient treated with inotersen.
Source: Journal of Cardiac Failure - Category: Cardiology Authors: Tags: 146 Source Type: research