Related Experiment Video
Updated: May 12, 2026

A Doxorubicin-induced Cardiomyopathy Model in Adult Zebrafish
Published on: June 7, 2018
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.
Abstract:
There is an unmet medical need to treat patients with severe hypertrophic cardiomyopathy leading to heart failure and death in children carrying pathogenic activating variants in the RAS/mitogen-activated protein kinase pathway. A retrospective analysis of 61 patients provides evidence for decreased mortality and morbidity with improved cardiac status in patients with RASopathy with severe hypertrophic cardiomyopathy receiving mitogen-activated protein kinase kinase inhibition (n = 30) vs those with standard-of-care treatment (n = 31). Side effects were not life threatening and were manageable. The data presented suggest that personalized therapies targeting underlying signaling pathway abnormalities might be effective in critically ill patients with RASopathy warranting clinical investigation.
More Related Videos
03:45Investigating the Pathogenesis of MYH7 Mutation Gly823Glu in Familial Hypertrophic Cardiomyopathy using a Mouse Model
Published on: August 8, 2022
09:58An Efficient Protocol to Assess ERK Activity Modulation in Early Zebrafish Noonan Syndrome Models via Live FRET Microscopy and Immunofluorescence
Published on: May 2, 2025
Related Concept Videos
Mitogens and the Cell Cycle
Abnormal Proliferation
The Ras Gene
Ras is a...
mTOR Signaling and Cancer Progression
The mTOR pathway or the...
Small GTPases - Ras and Rho
Three regulatory proteins control their activity:
MAPK Signaling Cascades