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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Published inJACC. Case reports Vol. 29; no. 13; p. 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 Journal Article
LanguageEnglish
Published Netherlands Elsevier Inc 03.07.2024
Elsevier
Subjects
cardiovascular genetics
Case Report
hypertrophic cardiomyopathy
Noonan syndrome
RASopathies
cardiovascular genetics
hypertrophic cardiomyopathy
HCM
RASopathies
LVOT
Noonan syndrome
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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. [Display omitted]
ISSN:2666-0849
2666-0849
DOI:10.1016/j.jaccas.2024.102379