Article
Randomized trial of bilateral gene therapy injection for m.11778G>A MT-ND4 Leber optic neuropathy.
Brain : a journal of neurology - 19 Apr 2023
Newman Nancy J, Yu-Wai-Man Patrick, Subramanian Prem S, Moster Mark L, Wang An-Guor, Donahue Sean P, Leroy Bart P, Carelli Valerio, Biousse Valerie, Vignal-Clermont Catherine, Sergott Robert C, Sadun Alfredo A, Rebolleda Fernández Gema, Chwalisz Bart K, Banik Rudrani, Bazin Fabienne, Roux Michel, Cox Eric D, Taiel Magali, Sahel José-Alain
Abstract excerpt
Leber hereditary optic neuropathy (LHON) is an important example of mitochondrial blindness with the m.11778G>A mutation in the MT-ND4 gene being the most common disease-causing mtDNA variant worldwide. The REFLECT phase 3 pivotal study is a randomized, double-masked, placebo-controlled trial investigating the efficacy and safety of bilateral intravitreal injection of lenadogene nolparvovec in patients with a...
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