%0 Journal Article
%A Yu-Wai-Man, Patrick
%A Carelli, Valerio
%A Newman, Nancy J
%A Silva, Magda Joana
%A Linden, Aki
%A Van Stavern, Gregory
%A Szaflik, Jacek P
%A Banik, Rudrani
%A Lubiński, Wojciech
%A Pemp, Berthold
%A Liao, Yaping Joyce
%A Subramanian, Prem S
%A Misiuk-Hojło, Marta
%A Newman, Steven
%A Castillo, Lorena
%A Kocięcki, Jarosław
%A Levin, Marc H
%A Muñoz-Negrete, Francisco Jose
%A Yagan, Ali
%A Cherninkova, Sylvia
%A Katz, David
%A Meunier, Audrey
%A Votruba, Marcela
%A Korwin, Magdalena
%A Dziedziak, Jacek
%A Jurkutė, Neringa
%A Harvey, Joshua P
%A La Morgia, Chiara
%A Priglinger, Claudia
%A Llòria, Xavier
%A Tomasso, Livia
%A Klopstock, Thomas
%T Therapeutic benefit of idebenone in patients with Leber hereditary optic neuropathy: The LEROS nonrandomized controlled trial.
%J Cell reports / Medicine
%V 5
%N 3
%@ 2666-3791
%C Maryland Heights, MO
%I Elsevier
%M DZNE-2024-00303
%P 101437
%D 2024
%X 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.
%K Humans
%K Optic Atrophy, Hereditary, Leber: drug therapy
%K Optic Atrophy, Hereditary, Leber: genetics
%K Optic Atrophy, Hereditary, Leber: diagnosis
%K Antioxidants: therapeutic use
%K Ubiquinone: therapeutic use
%K Ubiquinone: genetics
%K Ubiquinone: analogs & derivatives
%K Mutation
%K LHON (Other)
%K Leber hereditary optic neuropathy (Other)
%K idebenone (Other)
%K mitochondrial disease (Other)
%K mtDNA (Other)
%K neuro-ophthalmology (Other)
%K optic atrophy (Other)
%K optic neuropathy (Other)
%K retinal ganglion cells (Other)
%K idebenone (NLM Chemicals)
%K Antioxidants (NLM Chemicals)
%K Ubiquinone (NLM Chemicals)
%F PUB:(DE-HGF)16
%9 Journal Article
%2 pmc:PMC10982982
%$ pmid:38428428
%R 10.1016/j.xcrm.2024.101437
%U https://pub.dzne.de/record/268724