Translation of New and Emerging Therapies for Genetic Cardiomyopathies

The primary etiology of a diverse range of cardiomyopathies is now understood to be genetic, creating a new paradigm for targeting treatments on the basis of the underlying molecular cause. This review provides a genetic and etiologic context for the traditional clinical classifications of cardiomyopathy, including molecular subtypes that may exhibit differential responses to existing or emerging treatments. The authors describe several emerging cardiomyopathy treatments, including gene therapy, direct targeting of myofilament function, protein quality control, metabolism, and others. The authors discuss advantages and disadvantages of these approaches and indicate areas of high potential for short- and longer term efficacy. (C) 2022 The Authors. Published by Elsevier on behalf of the American College of Cardiology Foundation.

Automated summary

The primary cause of cardiomyopathies is now known to be genetic, leading to new approaches for treatment based on the underlying molecular cause. This review provides a genetic and etiological context for the traditional clinical classifications of cardiomyopathy and discusses emerging treatments that target specific molecular subtypes. The advantages and disadvantages of these approaches are described, and areas with high potential for short- and long-term efficacy are identified.