학술논문
Efficacy and safety of voretigene neparvovec (AAV2-hRPE65v2) in patients with RPE65-mediated inherited retinal dystrophy: a randomised, controlled, open-label, phase 3 trial
Document Type
Article
Author
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
Language
ISSN
0140-6736