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Impact of MEK Inhibition on Childhood RASopathy-Associated Hypertrophic Cardiomyopathy

Cordula M Wolf1, Martin Zenker2, Olga Boleti3

  • 1Department of Congenital Heart Defects and Pediatric Cardiology, German Heart Center Munich, School of Medicine and Health, Technical University of Munich, Munich, Germany; Deutsches Zentrum für Herz-Kreislauf-Forschung (German Center for Cardiovascular Research), partner site Munich Heart Alliance, Munich, Germany; Member of the European Reference Network for Rare and Low Prevalence Complex Diseases of the Heart.

JACC. Basic to Translational Science
|March 25, 2025
PubMed
Summary

Mitogen-activated protein kinase kinase inhibition shows promise in treating severe hypertrophic cardiomyopathy in children with RASopathy. This targeted therapy improved cardiac status and reduced mortality compared to standard care.

Keywords:
MEK inhibitionNoonan syndrome spectrumRAS/MAPK signalingRASopathieschildhood cardiomyopathysurvival

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