Treatment of RAF1-Related Obstructive Hypertrophic Cardiomyopathy by MEK Inhibition Using Trametinib

RASopathies cause nonsarcomeric hypertrophic cardiomyopathy via dysregulated signaling through RAS and upregulated mitogen-activated protein kinase activity. We provide the first report of the successful treatment of an adult with RAF1-associated hypertrophic cardiomyopathy using trametinib, a MEK i...

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Bibliographic Details
Published in:JACC. Case reports 2024, Vol.29 (13), p.102379-102379
Main Authors: Kiamanesh, Omid, Greenway, Steven C, Dicke, Franciscus, Ballantyne, Brennan, Mitrovic, Sasha, McGrath, Kaitlin, White, James A, Kent, William D T, Andelfinger, Gregor
Format: Report
Language:English
Online Access:Get full text
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Summary:RASopathies cause nonsarcomeric hypertrophic cardiomyopathy via dysregulated signaling through RAS and upregulated mitogen-activated protein kinase activity. We provide the first report of the successful treatment of an adult with RAF1-associated hypertrophic cardiomyopathy using trametinib, a MEK inhibitor.
ISSN:2666-0849
DOI:10.1016/j.jaccas.2024.102379