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As seen in: JAMA Cardiology

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AAVrh.10hFXN Gene Therapy for the Cardiomyopathy of Friedreich Ataxia : A Nonrandomized Clinical Trial

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By Ronald G. Crystal, Jonathan W. Weinsaft, Stephen M. Kaminsky, Anthony Caragiulo, Niamh Savage, Aarti Patel, Ralitza H Gavrilova, Susan Perlman, Madeline Galbraith, Udhay Krishnan, Robert J. Kaner, Abraham Sanders, Mary L. Vo, Harini Sarva, Dolan Sondhi, Bishnu P. De, Jason G. Mezey, Gregory Aubert, Aashir Khan, Nithya Selvan, Narinder Bhalla, Eric Adler Verified, Theresa A. Zesiewicz, Udhay Kahlon, Andrea Yoo
| JAMA Cardiology
AAVrh.10hFXN Gene Therapy for the Cardiomyopathy of Friedreich Ataxia: A Nonrandomized Clinical Trial Key Points Question Is gene therapy with an adenoassociated virus (AAV) coding for human frataxin a safe and preliminary effective therapy for the cardiomyopathy that characterizes Friedreich ataxia?
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