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Efficacy, Safety, and Durability of Voretigene Neparvovec-rzyl in RPE65 Mutation–Associated Inherited Retinal Dystrophy: Results of Phase 1 and 3 Trials
Albert M. Maguire ,
Stephen Russell ,
Jennifer A. Wellman ,
Daniel C. Chung ,
Zi-Fan Yu ,
Amy Tillman ,
Janet Wittes ,
Julie Pappas ,
Okan Elci ,
Kathleen A. Marshall ,
Sarah McCague ,
Hannah Reichert ,
Maria Davis ,
Francesca Simonelli ,
Bart P. Leroy ,
J. Fraser Wright ,
Katherine A. High ,
Jean Bennett
June 2019
June 2019
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