MEK Inhibitors for the Treatment of Hypertrophic Cardiomyopathy in Patients With RASopathies

Summary

The goal of this study is to evaluate the effectiveness of trametinib treatment in patients with Hyperthropic cardiomyopathy and a genetic mutation in the RAS/MAPK pathway.

Eligibility

Inclusion Criteria:

* patient with diagnosed RASopathy
* patient with diagnosed hypertrophic cardiomyopathy
* signed innform consent

Exclusion Criteria:

* contraindications to treatment with propranolol (drug hypersensitivity, atrioventricular block, severe bradycardia) disopyramide (drug hypersensitivity, WPW syndrome, atrioventricular block, QT prolongation) trametinib (drug hypersensitivity)
* lack of consent of the child's guardians to participate in the study

Conditions3

Interventions2

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