268724 20240403113254.0 pmc:PMC10982982 pmc 10.1016/j.xcrm.2024.101437 doi pmid:38428428 pmid altmetric:160241813 altmetric DZNE-2024-00303 English 610 Yu-Wai-Man, Patrick 0 Therapeutic benefit of idebenone in patients with Leber hereditary optic neuropathy: The LEROS nonrandomized controlled trial. Maryland Heights, MO 2024 Elsevier article DRIVER Output Types/Journal article DataCite Journal Article journal journal PUB:(DE-HGF)16 1711369101_22709 PUB:(DE-HGF) ARTICLE BibTeX JOURNAL_ARTICLE ORCID Journal Article 0 EndNote 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. 353 - Clinical and Health Care Research (POF4-353) G:(DE-HGF)POF4-353 POF4-353 POF IV 0 Dataset connected to CrossRef, PubMed, , Journals: pub.dzne.de 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 HB6PN45W4J NLM Chemicals Antioxidants NLM Chemicals Ubiquinone 1339-63-5 NLM Chemicals Humans MeSH Optic Atrophy, Hereditary, Leber: drug therapy MeSH Optic Atrophy, Hereditary, Leber: genetics MeSH Optic Atrophy, Hereditary, Leber: diagnosis MeSH Antioxidants: therapeutic use MeSH Ubiquinone: therapeutic use MeSH Ubiquinone: genetics MeSH Ubiquinone: analogs & derivatives MeSH Mutation MeSH Carelli, Valerio 1 Newman, Nancy J 2 Silva, Magda Joana 3 Linden, Aki 4 Van Stavern, Gregory 5 Szaflik, Jacek P 6 Banik, Rudrani 7 Lubiński, Wojciech 8 Pemp, Berthold 9 Liao, Yaping Joyce 10 Subramanian, Prem S 11 Misiuk-Hojło, Marta 12 Newman, Steven 13 Castillo, Lorena 14 Kocięcki, Jarosław 15 Levin, Marc H 16 Muñoz-Negrete, Francisco Jose 17 Yagan, Ali 18 Cherninkova, Sylvia 19 Katz, David 20 Meunier, Audrey 21 Votruba, Marcela 22 Korwin, Magdalena 23 Dziedziak, Jacek 24 Jurkutė, Neringa 25 Harvey, Joshua P 26 La Morgia, Chiara 27 Priglinger, Claudia 28 Llòria, Xavier 29 Tomasso, Livia 30 Klopstock, Thomas P:(DE-2719)2810704 31 Last author dzne Group, LEROS Study 32 Collaboration Author 10.1016/j.xcrm.2024.101437 Vol. 5, no. 3, p. 101437 - PERI:(DE-600)3019420-9 3 101437 Cell reports / Medicine 5 2024 2666-3791 http://pub.dzne.de/record/268724/files/DZNE-2024-00303%20SUP.zip OpenAccess http://pub.dzne.de/record/268724/files/DZNE-2024-00303.pdf OpenAccess pdfa http://pub.dzne.de/record/268724/files/DZNE-2024-00303.pdf?subformat=pdfa oai:pub.dzne.de:268724 openaire open_access VDB driver dnbdelivery Deutsches Zentrum für Neurodegenerative Erkrankungen I:(DE-588)1065079516 DZNE 31 P:(DE-2719)2810704 DE-HGF Gesundheit Neurodegenerative Diseases G:(DE-HGF)POF4-350 G:(DE-HGF)POF4-353 G:(DE-HGF)POF4 G:(DE-HGF)POF4-300 G:(DE-HGF)POF Clinical and Health Care Research 0 2024 DBCoverage StatID:(DE-HGF)0200 StatID SCOPUS 2023-08-22 DBCoverage StatID:(DE-HGF)0160 StatID Essential Science Indicators 2023-08-22 DBCoverage StatID:(DE-HGF)1050 StatID BIOSIS Previews 2023-08-22 DBCoverage StatID:(DE-HGF)1190 StatID Biological Abstracts 2023-08-22 Creative Commons Attribution CC BY 4.0 LIC:(DE-HGF)CCBY4 HGFVOC JCR StatID:(DE-HGF)0100 StatID CELL REP MED : 2022 2023-08-22 DBCoverage StatID:(DE-HGF)1030 StatID Current Contents - Life Sciences 2023-08-22 IF >= 10 StatID:(DE-HGF)9910 StatID CELL REP MED : 2022 2023-08-22 DBCoverage StatID:(DE-HGF)0501 StatID DOAJ Seal 2023-04-12T14:51:29Z DBCoverage StatID:(DE-HGF)0500 StatID DOAJ 2023-04-12T14:51:29Z WoS StatID:(DE-HGF)0113 StatID Science Citation Index Expanded 2023-08-22 Fees StatID:(DE-HGF)0700 StatID 2023-08-22 DBCoverage StatID:(DE-HGF)0150 StatID Web of Science Core Collection 2023-08-22 DBCoverage StatID:(DE-HGF)1110 StatID Current Contents - Clinical Medicine 2023-08-22 OpenAccess StatID:(DE-HGF)0510 StatID Peer Review StatID:(DE-HGF)0030 StatID DOAJ : Anonymous peer review 2023-04-12T14:51:29Z Article Processing Charges StatID:(DE-HGF)0561 StatID 2023-08-22 DBCoverage StatID:(DE-HGF)0300 StatID Medline 2023-08-22 DBCoverage StatID:(DE-HGF)0320 StatID PubMed Central 2023-08-22 DBCoverage StatID:(DE-HGF)0199 StatID Clarivate Analytics Master Journal List 2023-08-22 I:(DE-2719)1111015 Clinical Research (Munich) Clinical Research (Munich) 0 journal VDB UNRESTRICTED I:(DE-2719)1111015 FullTexts