These data identify both minimally and significantly effective therapeutic doses that are clinically relevant for the treatment of the cardiac manifestations of FA.
Friday, May 12, 2023
Identification of Safe and Effective Intravenous Dose of AAVrh.10hFXN to Treat the Cardiac Manifestations of Friedreich's Ataxia
Dr. Carlos Munoz-Zuluaga, Dr. Monica Gertz, Ms. Melissa Yost-Bido, Ms. Alessandria Greco, Mr. Nicholas Gorman, Mr. Alvin Chen, Mr. Vikrum Kooner, Dr. Jonathan B Rosenberg, Dr. Bishnu P De, Dr. Stephen M. Kaminsky, Dr. Alain Bborczuk, Dr. Rodolfo Ricart Arbona, Dr. Heather R Martin, Dr. Sebastien Monette, Dr. Richie Khanna, Dr. Jay A Barth, Dr. Ronald G Crystal, and Dr. Dolan Sondhi. Identification of Safe and Effective Intravenous Dose of AAVrh.10hFXN to Treat the Cardiac Manifestations of Friedreich's Ataxia. Human Gene Therapy. ahead of print doi:10.1089/hum.2023.020 Online Ahead of Editing: May 11, 2023
