Durability of Voretigene Neparvovec for Biallelic RPE65-Mediated Inherited Retinal Disease: Phase 3 Results at 3 and 4 Years.

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Abstract

To determine whether functional vision and visual function improvements after voretigene neparvovec (VN; Luxturna [Spark Therapeutics, Inc]) administration in patients with biallelic RPE65 mutation-associated inherited retinal disease are maintained at 3 to 4 years and to review safety outcomes.

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Journal ID (iso-abbrev): Ophthalmology

Title: Ophthalmology

Publisher: Elsevier BV

ISSN (Electronic): 1549-4713

ISSN (Print): 0161-6420

Publication date (Electronic): October 2021

Volume: 128

Issue: 10

Affiliations

[1 ] 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.

[2 ] Department of Ophthalmology and Visual Sciences, University of Iowa Institute for Vision Research, University of Iowa, Iowa City, Iowa.

[3 ] Spark Therapeutics, Inc., Philadelphia, Pennsylvania.

[4 ] SparingVision, Philadelphia, Pennsylvania.

[5 ] Multidisciplinary Department of Medical, Surgical and Dental Sciences, University of Campania "Luigi Vanvitelli", Naples, Italy.

[6 ] Division of Ophthalmology and Center for Cellular and Molecular Therapeutics, Children's Hospital of Philadelphia, Philadelphia, Pennsylvania; Department of Ophthalmology and Center for Medical Genetics Ghent, Ghent University Hospital, Ghent, Belgium.

[7 ] 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.

Article

Publisher Item ID: S0161-6420(21)00236-0

DOI: 10.1016/j.ophtha.2021.03.031

PubMed ID: 33798654

SO-VID: 3cacd96f-d462-4e19-91a9-95ba3109568b

History

Keywords: RPE65,gene augmentation,inherited retinal disease,voretigene neparvovec

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Keywords: RPE65, gene augmentation, inherited retinal disease, voretigene neparvovec

Durability of Voretigene Neparvovec for Biallelic RPE65-Mediated Inherited Retinal Disease: Phase 3 Results at 3 and 4 Years.

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Karger: Ophthalmology

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