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Efficacy, Safety, and Durability of Voretigene Neparvovec-rzyl in RPE65 Mutation-Associated Inherited Retinal Dystrophy: Results of Phase 1 and 3 Trials.
Maguire, Albert M; Russell, Stephen; Wellman, Jennifer A; Chung, Daniel C; Yu, Zi-Fan; Tillman, Amy; Wittes, Janet; Pappas, Julie; Elci, Okan; Marshall, Kathleen A; McCague, Sarah; Reichert, Hannah; Davis, Maria; Simonelli, Francesca; Leroy, Bart P; Wright, J Fraser; High, Katherine A; Bennett, Jean.
Afiliação
  • Maguire AM; Department of Ophthalmology, Perelman School of Medicine, University of Pennsylvania, Philadelphia, Pennsylvania; Center for Cellular and Molecular Therapeutics, Inc., Children's Hospital of Philadelphia, Philadelphia, Pennsylvania. Electronic address: amaguire@pennmedicine.upenn.edu.
  • Russell S; Department of Ophthalmology and Visual Sciences, University of Iowa Institute for Vision Research, University of Iowa, Iowa City, Iowa.
  • Wellman JA; Center for Cellular and Molecular Therapeutics, Inc., Children's Hospital of Philadelphia, Philadelphia, Pennsylvania; Spark Therapeutics, Inc., Philadelphia, Pennsylvania.
  • Chung DC; Center for Cellular and Molecular Therapeutics, Inc., Children's Hospital of Philadelphia, Philadelphia, Pennsylvania; Spark Therapeutics, Inc., Philadelphia, Pennsylvania.
  • Yu ZF; Statistics Collaborative, Inc., Washington, DC.
  • Tillman A; Statistics Collaborative, Inc., Washington, DC.
  • Wittes J; Statistics Collaborative, Inc., Washington, DC.
  • Pappas J; Westat Biostatistics and Data Management Core, Children's Hospital of Philadelphia, Philadelphia, Pennsylvania.
  • Elci O; Westat Biostatistics and Data Management Core, Children's Hospital of Philadelphia, Philadelphia, Pennsylvania.
  • Marshall KA; Center for Cellular and Molecular Therapeutics, Inc., Children's Hospital of Philadelphia, Philadelphia, Pennsylvania.
  • McCague S; Center for Cellular and Molecular Therapeutics, Inc., Children's Hospital of Philadelphia, Philadelphia, Pennsylvania.
  • Reichert H; University of Iowa Health Care, Iowa City, Iowa.
  • Davis M; University of Iowa Health Care, Iowa City, Iowa.
  • Simonelli F; 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, Inc., Children's Hospital of Philadelphia, Philadelphia, Pennsylvania; Department of Ophthalmology and Center for Medical Genetics Ghent, Ghent University Hospital, Ghent, Belgium.
  • Wright JF; Center for Cellular and Molecular Therapeutics, Inc., Children's Hospital of Philadelphia, Philadelphia, Pennsylvania; Spark Therapeutics, Inc., Philadelphia, Pennsylvania.
  • High KA; Center for Cellular and Molecular Therapeutics, Inc., Children's Hospital of Philadelphia, Philadelphia, Pennsylvania; Spark Therapeutics, Inc., Philadelphia, Pennsylvania.
  • Bennett J; Department of Ophthalmology, Perelman School of Medicine, University of Pennsylvania, Philadelphia, Pennsylvania; Center for Cellular and Molecular Therapeutics, Inc., Children's Hospital of Philadelphia, Philadelphia, Pennsylvania.
Ophthalmology ; 126(9): 1273-1285, 2019 09.
Article em En | MEDLINE | ID: mdl-31443789
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Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Assunto principal: Terapia Genética / Dependovirus / Cis-trans-Isomerases / Distrofias Retinianas / Vetores Genéticos / Mutação Tipo de estudo: Clinical_trials / Observational_studies / Prognostic_studies / Risk_factors_studies Limite: Adolescent / Adult / Child / Female / Humans / Male Idioma: En Ano de publicação: 2019 Tipo de documento: Article
Texto completo
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Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Assunto principal: Terapia Genética / Dependovirus / Cis-trans-Isomerases / Distrofias Retinianas / Vetores Genéticos / Mutação Tipo de estudo: Clinical_trials / Observational_studies / Prognostic_studies / Risk_factors_studies Limite: Adolescent / Adult / Child / Female / Humans / Male Idioma: En Ano de publicação: 2019 Tipo de documento: Article