Treatment For Filamin C-Related Cardiomyopathies


­Filamin C (FLNC) mutations have been identified as a genetic cause of dilated cardiomyopathy (DCM) with an arrhythmogenic phenotype and an increased risk for sudden cardiac death due to ventricular arrhythmias. Many of these patients also develop heart failure, requiring heart transplant. Unfortunately, there are currently no specific treatments for DCM due to FLNC mutations. A team led by Dr. Suet Nee Chen has discovered that Crenolanib may be able to to improve contractile function and decrease arrhythmogenic potential in patients with FLNC mutant DCM. In vitro studies suggest that PDGFRA inhibition with Crenolanib improves contractile function and reduces arrythmias in patient iPSC cardiomyocytes. A therapeutic for the treatment of FLNC mutant DCM may improve life for thousands of affected individuals in the U.S. through a reduction in fatal arrythmias and preventing or delaying cardiac transplant.

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For Information, Contact:
Mary Tapolsky
University of Colorado
Suet Nee Chen
Luisa Mestroni
Joseph Wu
Matthew R.G. Taylor
Ying Wooi Wan
Chi Keung Lam
Disease Areas:
Regenerative Medicine
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