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Article

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

Ophthalmology - 1 Oct 2021

Maguire Albert M, Russell Stephen, Chung Daniel C, Yu Zi-Fan, Tillman Amy, Drack Arlene V, Simonelli Francesca, Leroy Bart P, Reape Kathleen Z, High Katherine A, Bennett Jean

Abstract excerpt

PURPOSE: 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. DESIGN: Open-label, randomized, controlled phase 3 trial. PARTICIPANTS: Thirty-one individuals were enrolled...

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PMID: 33798654
DOI: 10.1016/j.ophtha.2021.03.031

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