HCM

This project seeks to perform a Phase 3 clinical trial to test whether an FDA-approved cancer drug called trametinib is effective in treating young infants with genetic conditions called RASopathies and a severe, life-threatening heart problem called hypertrophic cardiomyopathy. The purpose of this research is to demonstrate that trametinib is effective in preventing these sick babies from dying, receiving a heart transplant or undergoing heart surgery to remove extra heart muscle over a one-year period. In addition, the investigators will determine how often this heart problem comes back when the trametinib treatment is stopped.

This study aimed to evaluate the efficacy of non-invasive dynamic blood pressure monitoring in assessing left ventricular outflow tract obstruction in patients with hypertrophic cardiomyopathy. We prospectively collected SBP waveforms and LVOT gradient data from patients with obstructive and non-obstructive disease, systematically analyzed their correlation, and explored its clinical value in different clinical scenarios.