Emerging Therapies for Transthyretin Cardiac Amyloidosis. Current treatment options in cardiovascular medicine Alexander, K. M., Evangelisti, A. n., Witteles, R. M. 2019; 21 (8): 40


Transthyretin cardiac amyloidosis is an underdiagnosed, undertreated disease which is associated with significant morbidity and mortality. This review will discuss the recent advancements in novel therapies for transthyretin amyloidosis.In recent phase 3 clinical trials, transthyretin stabilizers (tafamidis) and transthyretin silencers (patisiran and inotersen) have proven to be effective therapies for various forms of transthyretin amyloidosis. Understanding the recent and upcoming clinical trials for transthyretin amyloidosis will be important for improving the management of this challenging disease.

View details for DOI 10.1007/s11936-019-0743-2

View details for PubMedID 31309347