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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.
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.
Afiliación
  • Russell S; Department of Ophthalmology and Visual Sciences, University of Iowa, Iowa City, IA, USA. Electronic address: steve-russell@uiowa.edu.
  • Bennett J; Department of Ophthalmology, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USA.
  • Wellman JA; Spark Therapeutics, Philadelphia, PA, USA.
  • Chung DC; Spark Therapeutics, Philadelphia, PA, USA.
  • Yu ZF; Statistics Collaborative, Washington, DC, USA.
  • Tillman A; Statistics Collaborative, Washington, DC, USA.
  • Wittes J; Statistics Collaborative, Washington, DC, USA.
  • Pappas J; Westat Biostatistics and Data Management Core, Children's Hospital of Philadelphia, Philadelphia, PA, USA.
  • Elci O; Westat Biostatistics and Data Management Core, Children's Hospital of Philadelphia, Philadelphia, PA, USA.
  • McCague S; Center for Cellular and Molecular Therapeutics, Children's Hospital of Philadelphia, Philadelphia, PA, USA.
  • Cross D; Center for Cellular and Molecular Therapeutics, Children's Hospital of Philadelphia, Philadelphia, PA, USA.
  • Marshall KA; Center for Cellular and Molecular Therapeutics, Children's Hospital of Philadelphia, Philadelphia, PA, USA.
  • Walshire J; University of Iowa Health Care, Iowa City, Iowa, USA.
  • Kehoe TL; University of Iowa Health Care, Iowa City, Iowa, USA.
  • Reichert H; University of Iowa Health Care, Iowa City, Iowa, USA.
  • Davis M; University of Iowa Health Care, Iowa City, Iowa, USA.
  • Raffini L; Department of Hematology, Children's Hospital of Philadelphia, Philadelphia, PA, USA.
  • George LA; Department of Hematology, Children's Hospital of Philadelphia, Philadelphia, PA, USA.
  • Hudson FP; 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.
  • Dingfield L; Division of General Internal Medicine, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USA.
  • Zhu X; Department of Hematology, Children's Hospital of Philadelphia, Philadelphia, PA, USA.
  • Haller JA; Wills Eye Hospital and Department of Ophthalmology, Jefferson Medical College, Thomas Jefferson University and Thomas Jefferson University Hospitals, Philadelphia, PA, USA.
  • Sohn EH; Department of Ophthalmology and Visual Sciences, University of Iowa, Carver College of Medicine, Iowa City, IA, USA.
  • Mahajan VB; Department of Ophthalmology and Visual Sciences, University of Iowa, Carver College of Medicine, Iowa City, IA, USA.
  • Pfeifer W; University of Iowa Health Care, Iowa City, Iowa, USA.
  • Weckmann M; Department of Psychiatry, University of Iowa, Carver College of Medicine, Iowa City, IA, USA.
  • Johnson C; Department of Ophthalmology and Visual Sciences, University of Iowa, Carver College of Medicine, Iowa City, IA, USA.
  • Gewaily D; Philadelphia Retina Associates, Philadelphia, PA, USA.
  • Drack A; Department of Ophthalmology and Visual Sciences, University of Iowa, Carver College of Medicine, Iowa City, IA, USA.
  • Stone E; Department of Ophthalmology and Visual Sciences, University of Iowa, Iowa City, IA, USA.
  • Wachtel K; Spark Therapeutics, Philadelphia, PA, USA.
  • Simonelli F; Eye Clinic, Multidisciplinary Department of Medical, Surgical and Dental Sciences, University of Campania Luigi Vanvitelli, Naples, Italy.
  • Leroy BP; 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.
  • Wright JF; Spark Therapeutics, Philadelphia, PA, USA.
  • High KA; Spark Therapeutics, Philadelphia, PA, USA.
  • Maguire AM; Department of Ophthalmology, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USA.
Lancet ; 390(10097): 849-860, 2017 Aug 26.
Article en En | MEDLINE | ID: mdl-28712537
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Texto completo: 1 Colección: 01-internacional Base de datos: MEDLINE Asunto principal: Terapia Genética / Cis-trans-Isomerasas / Distrofias Retinianas Tipo de estudio: Clinical_trials Límite: Adolescent / Female / Humans / Male País/Región como asunto: America do norte Idioma: En Revista: Lancet Año: 2017 Tipo del documento: Article Pais de publicación: Reino Unido
Texto completo
Añadir a Mi BVS
Imprimir
XML
PubMed Links
Buscar en Google

Texto completo: 1 Colección: 01-internacional Base de datos: MEDLINE Asunto principal: Terapia Genética / Cis-trans-Isomerasas / Distrofias Retinianas Tipo de estudio: Clinical_trials Límite: Adolescent / Female / Humans / Male País/Región como asunto: America do norte Idioma: En Revista: Lancet Año: 2017 Tipo del documento: Article Pais de publicación: Reino Unido