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8. Efficacy, Safety, and Durability of Voretigene Neparvovec-rzyl in RPE65 Mutation-Associated Inherited Retinal Dystrophy: Results of Phase 1 and 3 Trials. Maguire AM; Russell S; Wellman JA; Chung DC; Yu ZF; Tillman A; Wittes J; Pappas J; Elci O; Marshall KA; McCague S; Reichert H; Davis M; Simonelli F; Leroy BP; Wright JF; High KA; Bennett J Ophthalmology; 2019 Sep; 126(9):1273-1285. PubMed ID: 31443789 [TBL] [Abstract][Full Text] [Related]
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13. Voretigene Neparvovec: A Review in RPE65 Mutation-Associated Inherited Retinal Dystrophy. Kang C; Scott LJ Mol Diagn Ther; 2020 Aug; 24(4):487-495. PubMed ID: 32535767 [TBL] [Abstract][Full Text] [Related]
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19. The Natural History of Inherited Retinal Dystrophy Due to Biallelic Mutations in the RPE65 Gene. Chung DC; Bertelsen M; Lorenz B; Pennesi ME; Leroy BP; Hamel CP; Pierce E; Sallum J; Larsen M; Stieger K; Preising M; Weleber R; Yang P; Place E; Liu E; Schaefer G; DiStefano-Pappas J; Elci OU; McCague S; Wellman JA; High KA; Reape KZ Am J Ophthalmol; 2019 Mar; 199():58-70. PubMed ID: 30268864 [TBL] [Abstract][Full Text] [Related]
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