Article

Rigosertib Reverses Hypertrophic Cardiomyopathy in <i>RAF1</i> -Associated Noonan Syndrome

2024-11-25

Abstract excerpt

<h4>Background</h4> RASopathies constitute a group of rare genetic disorders caused by mutations in genes that reside along the canonical Ras/MAPK signaling pathway, affecting cell growth and differentiation. These syndromes, which include conditions like Noonan syndrome (NS), are characterized by developmental delays, distinctive facial dysmorphia, and a variety of cardiac defects, notably hypertrophic cardiomyo...

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Literature Corpus work: 39d77f2a-7496-5150-b862-6ad192bbc656
DOI: 10.1101/2024.11.25.623865

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