140 Gene therapy for Friedreich ataxia cardiomyopathy: safety and preliminary assessment of efficacy; Ronald G. Crystal, Jonathan W. Weinsaft, Stephen M. Kaminsky, Anthony Caragiulo, Aarti Patel, Ralitza H. Gavrilova, Susan L. Perlman, Udhay Krishnan, Madeline Galbraith, Niamh Savage, Robert J. Kaner, Abraham Sanders, Mary Vo, Harini Sarva, Andrea Yoo, Dolan Sondhi, Bishnu P. De, Gregory Aubert, Aashir Khan, Nithya Selvan, Sandi See Tai, Narinder Bhalla, Eric Adler, Theresa Zesiewicz.
This interim data suggests that a single intravenous administration of AAVrh.10hFXN to
individuals with FA cardiomyopathy is generally safe and biologically active, increasing cardiac FXN expression, improving a key marker of cardiac structure, reducing a serum biomarker relevant to cardiomyocyte integrity and improving or stabilizing neurologic function.
