Trametinib shows promise in treating severe hypertrophic cardiomyopathy in children
Raluca Maniță
American College of Cardiology Jan 8 2025 Trametinib, a mitogen-activated protein kinase (MEK) inhibitor, reduces mortality and morbidity in children with severe hypertrophic cardiomyopathy (HCM) caused by pathogenic variants in the RAS/MAPK pathway, according to a study published today in JACC: Basic to Translational Science . The study provides strong evidence for personalized treatment targeting the underlying genetic causes of RASopathies, a group of rare disorders that often lead to life-
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