Risedronate 💊: A New Hope for Genetic Cardiomyopathy ❤️
Dr RR Baliga's "Got Knowledge Doc" Podkast
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Show Notes
The study investigates the K210del mutation in genetic cardiomyopathy, which impairs myocardial contractility due to calcium discoordination in the troponin complex.
Using a structure-based drug repurposing approach, researchers identified risedronate, an FDA-approved bisphosphonate, as a structural corrector that restores the calcium-binding domain's normal configuration.
Experiments with patient-derived iPSC cardiomyocytes and a mouse model demonstrated that risedronate normalized calcium sensitivity, improved contraction velocity, and restored left ventricular function.
The findings highlight the potential of repurposing existing drugs to treat genetic cardiomyopathies.