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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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Principais autores: Omid Kiamanesh, MD, Steven C. Greenway, MD, Franciscus Dicke, MD, MBA, Brennan Ballantyne, MD, Sasha Mitrovic, RDCS, Kaitlin McGrath, MD, James A. White, MD, William D.T. Kent, MD, Gregor Andelfinger, MD
Formato: Artigo
Idioma:Inglês
Publicado em: Elsevier 2024-07-01
coleção:JACC: Case Reports
Assuntos:
cardiovascular genetics
hypertrophic cardiomyopathy
Noonan syndrome
RASopathies
Acesso em linha:http://www.sciencedirect.com/science/article/pii/S2666084924001724
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