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

doi:10.1016/S0140-6736(17)31868-8

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Article

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

Lancet (London, England) - 26 Aug 2017

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

Abstract excerpt

BACKGROUND: Phase 1 studies have shown potential benefit of gene replacement in RPE65-mediated inherited retinal dystrophy. This phase 3 study assessed the efficacy and safety of voretigene neparvovec in participants whose inherited retinal dystrophy would otherwise progress to complete blindness. METHODS: In this open-label, randomised, controlled phase 3 trial done at two sites in the USA, individuals aged 3...

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Identifiers

PMID: 28712537
DOI: 10.1016/S0140-6736(17)31868-8
PMCID: PMC5726391

Genes

RPE65

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