Emerging Targeted Therapies for Inherited Cardiomyopathies and Arrhythmias

Inherited cardiomyopathy and arrhythmia syndromes are associated with significant morbidity and mortality, particularly in young people. Medical management of these conditions has primarily been limited to agents previously developed for more common forms of heart disease and not tailored to their distinct pathophysiology. As our understanding of their underlying genetics and disease mechanisms has improved, an era of targeted therapies for these rare conditions has begun to emerge. In recent years, several novel agents have been developed and tested in preclinical models and, in some cases, have advanced to both the clinical trial and clinical approval stages with exciting results. These new treatments are derived from multiple classes of therapeutics, including small molecules, antisense oligonucleotides, small interfering RNAs, adeno-associated virus–mediated gene therapies, and in vivo gene editing. Collectively, they carry the promise of revolutionizing management of affected patients and their families.