Efficacy and safety of voretigene neparvovec (AAV2-hRPE65v2) in patients with RPE65-mediated inherited retinal dystrophy: a randomised, controlled, open-label, phase 3 trial
Titel:
Efficacy and safety of voretigene neparvovec (AAV2-hRPE65v2) in patients with RPE65-mediated inherited retinal dystrophy: a randomised, controlled, open-label, phase 3 trial
Auteur:
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