17 Yu-Wai-Man, Patrick Carelli, Valerio Newman, Nancy J Silva, Magda Joana Linden, Aki Van Stavern, Gregory Szaflik, Jacek P Banik, Rudrani Lubiński, Wojciech Pemp, Berthold Liao, Yaping Joyce Subramanian, Prem S Misiuk-Hojło, Marta Newman, Steven Castillo, Lorena Kocięcki, Jarosław Levin, Marc H Muñoz-Negrete, Francisco Jose Yagan, Ali Cherninkova, Sylvia Katz, David Meunier, Audrey Votruba, Marcela Korwin, Magdalena Dziedziak, Jacek Jurkutė, Neringa Harvey, Joshua P La Morgia, Chiara Priglinger, Claudia Llòria, Xavier Tomasso, Livia Klopstock, Thomas Group, LEROS Study Clinical Research (Munich) Cell reports / Medicine Cell reports / Medicine Elsevier Maryland Heights, MO 2666-3791 10.1016/j.xcrm.2024.101437 English 101437 3 5 Leber hereditary optic neuropathy (LHON) is a mitochondrial disease leading to rapid and severe bilateral vision loss. Idebenone has been shown to be effective in stabilizing and restoring vision in patients treated within 1 year of onset of vision loss. The open-label, international, multicenter, natural history-controlled LEROS study (ClinicalTrials.gov NCT02774005) assesses the efficacy and safety of idebenone treatment (900 mg/day) in patients with LHON up to 5 years after symptom onset (N = 199) and over a treatment period of 24 months, compared to an external natural history control cohort (N = 372), matched by time since symptom onset. LEROS meets its primary endpoint and confirms the long-term efficacy of idebenone in the subacute/dynamic and chronic phases; the treatment effect varies depending on disease phase and the causative mtDNA mutation. The findings of the LEROS study will help guide the clinical management of patients with LHON. PUB:(DE-HGF)16, ; 0, ; Humans Optic Atrophy, Hereditary, Leber: drug therapy Optic Atrophy, Hereditary, Leber: genetics Optic Atrophy, Hereditary, Leber: diagnosis Antioxidants: therapeutic use Ubiquinone: therapeutic use Ubiquinone: genetics Ubiquinone: analogs & derivatives Mutation LHON Leber hereditary optic neuropathy idebenone mitochondrial disease mtDNA neuro-ophthalmology optic atrophy optic neuropathy retinal ganglion cells idebenone Antioxidants Ubiquinone Journal Article 2024 DZNE-2024-00303 2024 pmc:PMC10982982 pmid:38428428 https://pub.dzne.de/record/268724 https://doi.org/10.1016/j.xcrm.2024.101437