ArticleCirculation2026
Rigosertib Reverses Hypertrophic Cardiomyopathy in Noonan Syndrome.
Article in Circulation, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Not yet cited in PubMed.
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The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
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22 authors.
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Abstract
backgroundRASopathies constitute a group of rare genetic disorders caused by mutations in genes along the canonical RAS/mitogen-activated protein kinase (MAPK) signaling pathway, affecting cell growth and differentiation. These syndromes, which include Noonan syndrome (NS), are characterized by developmental delays, distinctive facial dysmorphia, and cardiac defects, notably hypertrophic cardiomyopathy (HCM). Despite their prevalence and impact, therapeutic options for RASopathies remain limited. Rigosertib, a novel dual RAS/MAPK and phosphatidylinositol 3-kinase (PI3K)/AKT pathway inhibitor, is currently in clinical trials for treatment of melanoma and recessive dystrophic epidermolysis bullosa. Here, we identify rigosertib as a candidate therapy for RAF1-associated HCM.
methodsWe performed a drug screen of clinically relevant compounds in transgenic
resultsRigosertib was effective across a panel of transgenic
conclusionsTogether, our findings suggest that rigosertib normalizes and reverses RASopathy-associated HCM and other NS-associated syndromic features, supporting its development as a promising treatment for RAF1-associated HCM and, potentially, other RASopathy-dependent pathologies. This study not only highlights the therapeutic potential of rigosertib but also demonstrates the utility of an integrated approach using
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