Efficacy and safety of voretigene neparvovec (AAV2-hRPE65v2) in patients with RPE65 -mediated inherited retinal dystrophy: a randomised, controlled, open-label, phase 3 trial
Stephen R. Russell(University of Iowa), Jean Bennett(University of Pennsylvania), Jennifer Wellman(Spark Therapeutics (United States)), Daniel C. Chung(Spark Therapeutics (United States)), Zi-Fan Yu(Statistics Collaborative), Amy Tillman(Statistics Collaborative), Janet Wittes(Statistics Collaborative), Julie Pappas(Westat (United States)), Okan U. Elci(Westat (United States)), Sarah McCague(Children's Hospital of Philadelphia), Dominique Cross(Children's Hospital of Philadelphia), Kathleen Marshall(Children's Hospital of Philadelphia), Jean Walshire(University of Iowa Health Care), Taylor Kehoe(University of Iowa Health Care), Hannah Reichert(University of Iowa Health Care), Maria Davis(University of Iowa Health Care), Leslie Raffini(Children's Hospital of Philadelphia), Lindsey A. George(Children's Hospital of Philadelphia), Fleur Hudson(University of Pennsylvania), Laura Dingfield(University of Pennsylvania), Xiaosong Zhu(Children's Hospital of Philadelphia), Julia A. Haller(Thomas Jefferson University Hospital), Elliott H. Sohn(University of Iowa), Vinit B. Mahajan(University of Iowa), Wanda Pfeifer(University of Iowa Health Care), Michelle T. Weckmann(University of Iowa), Chris A. Johnson(University of Iowa), Dina Y. Gewaily(Retina Associates), Arlene V. Drack(University of Iowa), Edwin M. Stone(University of Iowa), Katie Wachtel(Spark Therapeutics (United States)), Francesca Simonelli(University of Campania "Luigi Vanvitelli"), Bart P. Leroy(Children's Hospital of Philadelphia), J. Fraser Wright(Spark Therapeutics (United States)), Katherine A. High(Spark Therapeutics (United States)), Albert M. Maguire(University of Pennsylvania)
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