engYu-Wai-Man, PatrickCarelli, ValerioLiao, Yaping JoyceSubramanian, Prem SMisiuk-Hojło, MartaNewman, StevenCastillo, LorenaKocięcki, JarosławLevin, Marc HMuñoz-Negrete, Francisco JoseYagan, AliCherninkova, SylviaNewman, Nancy JKatz, DavidMeunier, AudreyVotruba, MarcelaKorwin, MagdalenaDziedziak, JacekJurkutė, NeringaHarvey, Joshua PLa Morgia, ChiaraPriglinger, ClaudiaLlòria, XavierSilva, Magda JoanaTomasso, LiviaKlopstock, ThomasGroup, LEROS StudyLinden, AkiVan Stavern, GregorySzaflik, Jacek PBanik, RudraniLubiński, WojciechPemp, BertholdTherapeutic benefit of idebenone in patients with Leber hereditary optic neuropathy: The LEROS nonrandomized controlled trial.info:eu-repo/classification/ddc/610HumansOptic Atrophy, Hereditary, Leber: drug therapyOptic Atrophy, Hereditary, Leber: geneticsOptic Atrophy, Hereditary, Leber: diagnosisAntioxidants: therapeutic useUbiquinone: therapeutic useUbiquinone: geneticsUbiquinone: analogs & derivativesMutationLHONLeber hereditary optic neuropathyidebenonemitochondrial diseasemtDNAneuro-ophthalmologyoptic atrophyoptic neuropathyretinal ganglion cellsidebenoneAntioxidantsUbiquinoneLeber 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.Cell reports / Medicine 5(3), 101437 (2024). doi:10.1016/j.xcrm.2024.101437info:eu-repo/semantics/articleinfo:eu-repo/semantics/publishedVersionElsevier2024info:eu-repo/semantics/openAccessDEhttps://pub.dzne.de/record/268724https://pub.dzne.de/search?p=id:%22DZNE-2024-00303%22Researchersinfo:eu-repo/semantics/altIdentifier/issn/2666-3791info:eu-repo/semantics/altIdentifier/doi/10.1016/j.xcrm.2024.101437info:eu-repo/semantics/altIdentifier/pmid/pmid:38428428