Left ventricular noncompaction (LVNC) is a rare cardiac disorder with heart failure and high risk of thromboembolic events, arrhythmia, and sudden cardiac death1,2. The unmet medical need for LVNC treatment is the limited symptom relief medications, such as anti-congestive medications (diuretics, ACE inhibitors, and beta-blockers) and antiplatelet therapy to prevent thrombo-embolism1, for LVNC patients. Using LVNC-hiPSC-CM plateform to identify drugable target and the therapeutic drug through screening the repurposed drugs, we identified simvastatin as the effective therapeutic drug for the improvement of cardiac function in LVNC patients with TNNT2 mutation. Because LVNC can be caused by multi-gene mutations, it needs more effort to clarify the therapeutic efficacy of simvastatin in LVNC cardiomyocytes derived different LVNC patients carrying different genetic mutations.
