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This study identifies rigosertib, a dual RAS/MAPK and PI3K/AKT pathway inhibitor, as an effective therapy for hypertrophic cardiomyopathy (HCM) associated with Noonan syndrome (NS). Using both Drosophila and mammalian models, rigosertib significantly reversed cardiac hypertrophy and improved other NS-linked phenotypes, outperforming currently available MEK inhibitor therapy.
