New treatment option for severe hypertrophic cardiomyopathy in children shows promise.

A recent study published in JACC: Basic to Translational Science highlights the potential of trametinib, a MEK inhibitor, in reducing mortality and morbidity in children with severe hypertrophic cardiomyopathy (HCM) caused by genetic variants in the RAS/MAPK pathway. This personalized treatment approach shows promise in addressing the urgent medical needs of children with RASopathies, a group of rare disorders associated with life-threatening cardiac complications. The study, involving 61 pediatric patients, demonstrated a significant reduction in adverse outcomes in the trametinib group compared to standard care, emphasizing the importance of genotype-specific therapies for rare diseases like RAS-HCM. [Extracted from the article]