CardiologyAAVrh.10hFXNcardiomyopathy
AAVrh.10hFXN Gene Therapy for Friedreich Ataxia Cardiomyopathy: Safety and Preliminary Efficacy in a Nonrandomized Clinical Trial
By MedXY|Jul 1, 2026
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 advance