Emerging, novel gene-modulating therapies for transthyretin amyloid cardiomyopathy

Abstract excerpt

Transthyretin amyloid cardiomyopathy (ATTR-CM) is a progressive, life-threatening disease caused by the pathological deposition of misfolded transthyretin (TTR) protein in the myocardium, leading to restrictive cardiomyopathy and heart failure. While TTR stabilizers such as tafamidis and acoramidis are the only FDA-approved treatments, novel gene-modulating therapies are emerging as transformative approaches....

Topics

Share this publication in a Topic to start or enrich a Post.

• Share to Amyloidosis: Diagnosis, Treatment, Outcomes
• Share to Cellular transport and secretion
• Share to Peptidase Inhibition and Analysis