Article
Efficacy and Safety of Intravitreal Gene Therapy for Leber Hereditary Optic Neuropathy Treated within 6 Months of Disease Onset.
Ophthalmology - 1 May 2021
Newman Nancy J, Yu-Wai-Man Patrick, Carelli Valerio, Moster Mark L, Biousse Valerie, Vignal-Clermont Catherine, Sergott Robert C, Klopstock Thomas, Sadun Alfredo A, Barboni Piero, DeBusk Adam A, Girmens Jean François, Rudolph Günther, Karanjia Rustum, Taiel Magali, Blouin Laure, Smits Gerard, Katz Barrett, Sahel José-Alain
Abstract excerpt
PURPOSE: To evaluate the efficacy of a single intravitreal injection of rAAV2/2-ND4 in subjects with visual loss from Leber hereditary optic neuropathy (LHON). DESIGN: RESCUE is a multicenter, randomized, double-masked, sham-controlled, phase 3 clinical trial. PARTICIPANTS: Subjects with the m.11778G>A mitochondrial DNA mutation and vision loss ≤6 months from onset in 1 or both eyes were included. METHODS: Each...
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