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@ARTICLE{YuWaiMan:268724,
      author       = {Yu-Wai-Man, Patrick and Carelli, Valerio and Newman, Nancy
                      J and Silva, Magda Joana and Linden, Aki and Van Stavern,
                      Gregory and Szaflik, Jacek P and Banik, Rudrani and
                      Lubiński, Wojciech and Pemp, Berthold and Liao, Yaping
                      Joyce and Subramanian, Prem S and Misiuk-Hojło, Marta and
                      Newman, Steven and Castillo, Lorena and Kocięcki, Jarosław
                      and Levin, Marc H and Muñoz-Negrete, Francisco Jose and
                      Yagan, Ali and Cherninkova, Sylvia and Katz, David and
                      Meunier, Audrey and Votruba, Marcela and Korwin, Magdalena
                      and Dziedziak, Jacek and Jurkutė, Neringa and Harvey,
                      Joshua P and La Morgia, Chiara and Priglinger, Claudia and
                      Llòria, Xavier and Tomasso, Livia and Klopstock, Thomas},
      collaboration = {Group, LEROS Study},
      title        = {{T}herapeutic benefit of idebenone in patients with {L}eber
                      hereditary optic neuropathy: {T}he {LEROS} nonrandomized
                      controlled trial.},
      journal      = {Cell reports / Medicine},
      volume       = {5},
      number       = {3},
      issn         = {2666-3791},
      address      = {Maryland Heights, MO},
      publisher    = {Elsevier},
      reportid     = {DZNE-2024-00303},
      pages        = {101437},
      year         = {2024},
      abstract     = {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.},
      keywords     = {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
                      (Other) / Leber hereditary optic neuropathy (Other) /
                      idebenone (Other) / mitochondrial disease (Other) / mtDNA
                      (Other) / neuro-ophthalmology (Other) / optic atrophy
                      (Other) / optic neuropathy (Other) / retinal ganglion cells
                      (Other) / idebenone (NLM Chemicals) / Antioxidants (NLM
                      Chemicals) / Ubiquinone (NLM Chemicals)},
      cin          = {Clinical Research (Munich)},
      ddc          = {610},
      cid          = {I:(DE-2719)1111015},
      pnm          = {353 - Clinical and Health Care Research (POF4-353)},
      pid          = {G:(DE-HGF)POF4-353},
      typ          = {PUB:(DE-HGF)16},
      pmc          = {pmc:PMC10982982},
      pubmed       = {pmid:38428428},
      doi          = {10.1016/j.xcrm.2024.101437},
      url          = {https://pub.dzne.de/record/268724},
}