Efficacy of Tafamidis in Patients with Hereditary or Wild-Type Transthyretin Amyloid Cardiomyopathy: Further Results from the ATTR-ACT Trial

Transthyretin amyloid cardiomyopathy (ATTR-CM), an underdiagnosed, fatal disease caused by the deposition of transthyretin amyloid fibrils in the heart leading to heart failure, can be hereditary, due to mutations in the transthyretin gene, or wild-type. Tafamidis, a selective transthyretin stabilizer which prevents tetramer dissociation and amyloidogenesis, was recently shown to be an effective treatment for ATTR-CM patients in the international, multicenter, double-blind, placebo-controlled, randomized, Tafamidis in Transthyretin Cardiomyopathy Clinical Trial (ATTR-ACT).
Source: The Journal of Heart and Lung Transplantation - Category: Transplant Surgery Authors: Tags: 483 Source Type: research