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

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. M...

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Detalhes bibliográficos
Publicado no:Lancet
Main Authors: 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, Francesco, Leroy, Bart P, Wright, J Fraser, High, Katherine A, Maguire, Albert M
Formato: Artigo
Idioma:Inglês
Publicado em: 2017
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Article
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC5726391/
https://ncbi.nlm.nih.gov/pubmed/28712537
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/S0140-6736(17)31868-8
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