JAMA Cardiology

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Among adults with Friedreich ataxia cardiomyopathy, intravenous AAVrh.10hFXN gene therapy was well tolerated and associated with increased cardiac frataxin, reduced left ventricular mass index, and lower serum troponin I. ja.ma/4wvCdKr

AAVrh.10hFXN Gene Therapy for the Cardiomyopathy of Friedreich Ataxia

This nonrandomized clinical trial evaluates the safety and preliminary efficacy of administration of AAVrh.10hFXN to adults as gene therapy to treat Friedreich ataxia cardiomyopathy.

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