Friedreich ataxia (FRDA) is a genetic disorder and heart disease is the leading cause of premature death in FRDA patients. There is currently no treatment for FRDA heart disease. We will use patient-specific stem cells to create a FRDA heart disease model-in-a-dish to achieve a better understanding of disease development and progression. This study will establish a pre-clinical human model of FRDA heart disease, for discovery of new therapies and to facilitate pre-clinical trials.
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