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000268724 1001_ $$aYu-Wai-Man, Patrick$$b0
000268724 245__ $$aTherapeutic benefit of idebenone in patients with Leber hereditary optic neuropathy: The LEROS nonrandomized controlled trial.
000268724 260__ $$aMaryland Heights, MO$$bElsevier$$c2024
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000268724 520__ $$aLeber hereditary optic neuropathy (LHON) is a mitochondrial disease leading to rapid and severe bilateral vision loss. Idebenone has been shown to be effective in stabilizing and restoring vision in patients treated within 1 year of onset of vision loss. The open-label, international, multicenter, natural history-controlled LEROS study (ClinicalTrials.gov NCT02774005) assesses the efficacy and safety of idebenone treatment (900 mg/day) in patients with LHON up to 5 years after symptom onset (N = 199) and over a treatment period of 24 months, compared to an external natural history control cohort (N = 372), matched by time since symptom onset. LEROS meets its primary endpoint and confirms the long-term efficacy of idebenone in the subacute/dynamic and chronic phases; the treatment effect varies depending on disease phase and the causative mtDNA mutation. The findings of the LEROS study will help guide the clinical management of patients with LHON.
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000268724 650_7 $$2Other$$aLHON
000268724 650_7 $$2Other$$aLeber hereditary optic neuropathy
000268724 650_7 $$2Other$$aidebenone
000268724 650_7 $$2Other$$amitochondrial disease
000268724 650_7 $$2Other$$amtDNA
000268724 650_7 $$2Other$$aneuro-ophthalmology
000268724 650_7 $$2Other$$aoptic atrophy
000268724 650_7 $$2Other$$aoptic neuropathy
000268724 650_7 $$2Other$$aretinal ganglion cells
000268724 650_7 $$0HB6PN45W4J$$2NLM Chemicals$$aidebenone
000268724 650_7 $$2NLM Chemicals$$aAntioxidants
000268724 650_7 $$01339-63-5$$2NLM Chemicals$$aUbiquinone
000268724 650_2 $$2MeSH$$aHumans
000268724 650_2 $$2MeSH$$aOptic Atrophy, Hereditary, Leber: drug therapy
000268724 650_2 $$2MeSH$$aOptic Atrophy, Hereditary, Leber: genetics
000268724 650_2 $$2MeSH$$aOptic Atrophy, Hereditary, Leber: diagnosis
000268724 650_2 $$2MeSH$$aAntioxidants: therapeutic use
000268724 650_2 $$2MeSH$$aUbiquinone: therapeutic use
000268724 650_2 $$2MeSH$$aUbiquinone: genetics
000268724 650_2 $$2MeSH$$aUbiquinone: analogs & derivatives
000268724 650_2 $$2MeSH$$aMutation
000268724 7001_ $$aCarelli, Valerio$$b1
000268724 7001_ $$aNewman, Nancy J$$b2
000268724 7001_ $$aSilva, Magda Joana$$b3
000268724 7001_ $$aLinden, Aki$$b4
000268724 7001_ $$aVan Stavern, Gregory$$b5
000268724 7001_ $$aSzaflik, Jacek P$$b6
000268724 7001_ $$aBanik, Rudrani$$b7
000268724 7001_ $$aLubiński, Wojciech$$b8
000268724 7001_ $$aPemp, Berthold$$b9
000268724 7001_ $$aLiao, Yaping Joyce$$b10
000268724 7001_ $$aSubramanian, Prem S$$b11
000268724 7001_ $$aMisiuk-Hojło, Marta$$b12
000268724 7001_ $$aNewman, Steven$$b13
000268724 7001_ $$aCastillo, Lorena$$b14
000268724 7001_ $$aKocięcki, Jarosław$$b15
000268724 7001_ $$aLevin, Marc H$$b16
000268724 7001_ $$aMuñoz-Negrete, Francisco Jose$$b17
000268724 7001_ $$aYagan, Ali$$b18
000268724 7001_ $$aCherninkova, Sylvia$$b19
000268724 7001_ $$aKatz, David$$b20
000268724 7001_ $$aMeunier, Audrey$$b21
000268724 7001_ $$aVotruba, Marcela$$b22
000268724 7001_ $$aKorwin, Magdalena$$b23
000268724 7001_ $$aDziedziak, Jacek$$b24
000268724 7001_ $$aJurkutė, Neringa$$b25
000268724 7001_ $$aHarvey, Joshua P$$b26
000268724 7001_ $$aLa Morgia, Chiara$$b27
000268724 7001_ $$aPriglinger, Claudia$$b28
000268724 7001_ $$aLlòria, Xavier$$b29
000268724 7001_ $$aTomasso, Livia$$b30
000268724 7001_ $$0P:(DE-2719)2810704$$aKlopstock, Thomas$$b31$$eLast author$$udzne
000268724 7001_ $$aGroup, LEROS Study$$b32$$eCollaboration Author
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