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AAVrh.10hFXN Gene Therapy for Friedreich Ataxia Cardiomyopathy: Safety and Preliminary Efficacy in a Nonrandomized Clinical Trial
This review synthesizes evidence from a pioneering nonrandomized trial demonstrating that intravenous AAVrh.10hFXN gene therapy is safe and may improve cardiac outcomes in Friedreich ataxia cardiomyopathy, highlighting translational advances in precision cardiac gene therapy.