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| Home > Publications Database > Therapeutic benefit of idebenone in patients with Leber hereditary optic neuropathy: The LEROS nonrandomized controlled trial. > print |
| Home > Publications Database > Therapeutic benefit of idebenone in patients with Leber hereditary optic neuropathy: The LEROS nonrandomized controlled trial. > print |
| 001 | 268724 | ||
| 005 | 20240403113254.0 | ||
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| 037 | _ | _ | |a DZNE-2024-00303 |
| 041 | _ | _ | |a English |
| 082 | _ | _ | |a 610 |
| 100 | 1 | _ | |a Yu-Wai-Man, Patrick |b 0 |
| 245 | _ | _ | |a Therapeutic benefit of idebenone in patients with Leber hereditary optic neuropathy: The LEROS nonrandomized controlled trial. |
| 260 | _ | _ | |a Maryland Heights, MO |c 2024 |b Elsevier |
| 336 | 7 | _ | |a article |2 DRIVER |
| 336 | 7 | _ | |a Output Types/Journal article |2 DataCite |
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| 520 | _ | _ | |a 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. |
| 536 | _ | _ | |a 353 - Clinical and Health Care Research (POF4-353) |0 G:(DE-HGF)POF4-353 |c POF4-353 |f POF IV |x 0 |
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| 650 | _ | 7 | |a LHON |2 Other |
| 650 | _ | 7 | |a Leber hereditary optic neuropathy |2 Other |
| 650 | _ | 7 | |a idebenone |2 Other |
| 650 | _ | 7 | |a mitochondrial disease |2 Other |
| 650 | _ | 7 | |a mtDNA |2 Other |
| 650 | _ | 7 | |a neuro-ophthalmology |2 Other |
| 650 | _ | 7 | |a optic atrophy |2 Other |
| 650 | _ | 7 | |a optic neuropathy |2 Other |
| 650 | _ | 7 | |a retinal ganglion cells |2 Other |
| 650 | _ | 7 | |a idebenone |0 HB6PN45W4J |2 NLM Chemicals |
| 650 | _ | 7 | |a Antioxidants |2 NLM Chemicals |
| 650 | _ | 7 | |a Ubiquinone |0 1339-63-5 |2 NLM Chemicals |
| 650 | _ | 2 | |a Humans |2 MeSH |
| 650 | _ | 2 | |a Optic Atrophy, Hereditary, Leber: drug therapy |2 MeSH |
| 650 | _ | 2 | |a Optic Atrophy, Hereditary, Leber: genetics |2 MeSH |
| 650 | _ | 2 | |a Optic Atrophy, Hereditary, Leber: diagnosis |2 MeSH |
| 650 | _ | 2 | |a Antioxidants: therapeutic use |2 MeSH |
| 650 | _ | 2 | |a Ubiquinone: therapeutic use |2 MeSH |
| 650 | _ | 2 | |a Ubiquinone: genetics |2 MeSH |
| 650 | _ | 2 | |a Ubiquinone: analogs & derivatives |2 MeSH |
| 650 | _ | 2 | |a Mutation |2 MeSH |
| 700 | 1 | _ | |a Carelli, Valerio |b 1 |
| 700 | 1 | _ | |a Newman, Nancy J |b 2 |
| 700 | 1 | _ | |a Silva, Magda Joana |b 3 |
| 700 | 1 | _ | |a Linden, Aki |b 4 |
| 700 | 1 | _ | |a Van Stavern, Gregory |b 5 |
| 700 | 1 | _ | |a Szaflik, Jacek P |b 6 |
| 700 | 1 | _ | |a Banik, Rudrani |b 7 |
| 700 | 1 | _ | |a Lubiński, Wojciech |b 8 |
| 700 | 1 | _ | |a Pemp, Berthold |b 9 |
| 700 | 1 | _ | |a Liao, Yaping Joyce |b 10 |
| 700 | 1 | _ | |a Subramanian, Prem S |b 11 |
| 700 | 1 | _ | |a Misiuk-Hojło, Marta |b 12 |
| 700 | 1 | _ | |a Newman, Steven |b 13 |
| 700 | 1 | _ | |a Castillo, Lorena |b 14 |
| 700 | 1 | _ | |a Kocięcki, Jarosław |b 15 |
| 700 | 1 | _ | |a Levin, Marc H |b 16 |
| 700 | 1 | _ | |a Muñoz-Negrete, Francisco Jose |b 17 |
| 700 | 1 | _ | |a Yagan, Ali |b 18 |
| 700 | 1 | _ | |a Cherninkova, Sylvia |b 19 |
| 700 | 1 | _ | |a Katz, David |b 20 |
| 700 | 1 | _ | |a Meunier, Audrey |b 21 |
| 700 | 1 | _ | |a Votruba, Marcela |b 22 |
| 700 | 1 | _ | |a Korwin, Magdalena |b 23 |
| 700 | 1 | _ | |a Dziedziak, Jacek |b 24 |
| 700 | 1 | _ | |a Jurkutė, Neringa |b 25 |
| 700 | 1 | _ | |a Harvey, Joshua P |b 26 |
| 700 | 1 | _ | |a La Morgia, Chiara |b 27 |
| 700 | 1 | _ | |a Priglinger, Claudia |b 28 |
| 700 | 1 | _ | |a Llòria, Xavier |b 29 |
| 700 | 1 | _ | |a Tomasso, Livia |b 30 |
| 700 | 1 | _ | |a Klopstock, Thomas |0 P:(DE-2719)2810704 |b 31 |e Last author |u dzne |
| 700 | 1 | _ | |a Group, LEROS Study |b 32 |e Collaboration Author |
| 773 | _ | _ | |a 10.1016/j.xcrm.2024.101437 |g Vol. 5, no. 3, p. 101437 - |0 PERI:(DE-600)3019420-9 |n 3 |p 101437 |t Cell reports / Medicine |v 5 |y 2024 |x 2666-3791 |
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