Efficacy and safety of voretigene neparvovec (AAV2-hRPE65v2) in patients with RPE65-mediated inherited retinal dystrophy: a randomised, controlled, open-label, phase 3 trial
Resource Type
Article
Authors
Russell, Stephen ; Bennett, Jean ; Wellman, Jennifer A ; Chung, Daniel C ; Yu, Zi-Fan ; Tillman, Amy ; Wittes, Janet ; Pappas, Julie ; Elci, Okan ; McCague, Sarah ; Cross, Dominique ; Marshall, Kathleen A ; Walshire, Jean ; Kehoe, Taylor L ; Reichert, Hannah ; Davis, Maria ; Raffini, Leslie ; George, Lindsey A ; Hudson, F Parker ; Dingfield, Laura ; Zhu, Xiaosong ; Haller, Julia A ; Sohn, Elliott H ; Mahajan, Vinit B ; Pfeifer, Wanda ; Weckmann, Michelle ; Johnson, Chris ; Gewaily, Dina ; Drack, Arlene ; Stone, Edwin ; Wachtel, Katie ; Simonelli, Francesca ; Leroy, Bart P ; Wright, J Fraser ; High, Katherine A ; Maguire, Albert M
Source
In The Lancet 26 August-1 September 2017 390(10097):849-860
Subject
Primary Research Articles
Language
ISSN
0140-6736