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

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第一作者: Stephen,Russell
第一单位: Department of Ophthalmology and Visual Sciences, University of Iowa, Iowa City, IA, USA. Electronic address: steve-russell@uiowa.edu.
作者单位: Department of Ophthalmology and Visual Sciences, University of Iowa, Iowa City, IA, USA. Electronic address: steve-russell@uiowa.edu. [1] Department of Ophthalmology, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USA. [2] Spark Therapeutics, Philadelphia, PA, USA. [3] Statistics Collaborative, Washington, DC, USA. [4] Westat Biostatistics and Data Management Core, Children's Hospital of Philadelphia, Philadelphia, PA, USA. [5] Center for Cellular and Molecular Therapeutics, Children's Hospital of Philadelphia, Philadelphia, PA, USA. [6] University of Iowa Health Care, Iowa City, Iowa, USA. [7] Department of Hematology, Children's Hospital of Philadelphia, Philadelphia, PA, USA. [8] Department of Pediatrics, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USA; Department of Medicine, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USA. [9] Division of General Internal Medicine, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USA. [10] Wills Eye Hospital and Department of Ophthalmology, Jefferson Medical College, Thomas Jefferson University and Thomas Jefferson University Hospitals, Philadelphia, PA, USA. [11] Department of Ophthalmology and Visual Sciences, University of Iowa, Carver College of Medicine, Iowa City, IA, USA. [12] Department of Psychiatry, University of Iowa, Carver College of Medicine, Iowa City, IA, USA. [13] Philadelphia Retina Associates, Philadelphia, PA, USA. [14] Department of Ophthalmology and Visual Sciences, University of Iowa, Iowa City, IA, USA. [15] Eye Clinic, Multidisciplinary Department of Medical, Surgical and Dental Sciences, University of Campania Luigi Vanvitelli, Naples, Italy. [16] Division of Ophthalmology and Center for Cellular and Molecular Therapeutics, Children's Hospital of Philadelphia, Philadelphia, PA, USA; Department of Ophthalmology, Ghent University Hospital, Ghent, Belgium. [17]
DOI 10.1016/S0140-6736(17)31868-8
PMID 28712537
发布时间 2022-04-08
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