Indirect Comparison of Lenadogene Nolparvovec Gene Therapy Versus Natural History in Patients with Leber Hereditary Optic Neuropathy Carrying the m.11778G>A MT-ND4 Mutation.
Valerio CarelliNancy J NewmanPatrick Yu-Wai-ManValerie BiousseMark L MosterPrem S SubramanianCatherine Vignal-ClermontAn-Guor WangSean P DonahueBart P LeroyRobert C SergottThomas KlopstockAlfredo A SadunGema Rebolleda FernándezBart K ChwaliszRudrani BanikJean François GirmensChiara La MorgiaAdam A DeBuskNeringa JurkuteClaudia PriglingerRustum KaranjiaConstant JosseJulie SalzmannFrançois MontestrucMichel RouxMagali TaielJosé-Alain Sahelnull nullPublished in: Ophthalmology and therapy (2022)
NCT02652780 (REVERSE); NCT02652767 (RESCUE); NCT03406104 (RESTORE); NCT03293524 (REFLECT); NCT03295071 (REALITY).
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