Myosin inhibitor reverses hypertrophic cardiomyopathy in genotypically diverse pediatric iPSC-cardiomyocytes to mirror variant correction
Kinnear et al. demonstrate the ability of myosin inhibitors to restore function in pediatric hypertrophic cardiomyopathy patient and gene-edited iPSC-cardiomyocytes harboring myosin variants. From monogenic to digenic to biallelic variants, these inhibitors outperform non-targeted drugs, signaling a promising path for myosin-targeted therapies and an urgent call for clinical trials in children.