Efficacy and Safety of Intravitreal Gene Therapy for Leber Hereditary Optic Neuropathy Treated within 6 Months of Disease Onset.
Nancy J Newman,
Patrick Yu-Wai-Man,
Valerio Carelli,
Mark L Moster,
Valerie Biousse,
Catherine Vignal-Clermont,
Robert C Sergott,
Thomas Klopstock,
Alfredo A Sadun,
Piero Barboni,
Adam A DeBusk,
Jean François Girmens,
Günther Rudolph,
Rustum Karanjia,
Magali Taiel,
Laure Blouin,
Gerard Smits,
Barrett Katz,
José-Alain Sahel,
LHON Study Group
Apr 15, 2021
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...