| Identifier |
20240304_nanos_global1_04 |
| Title |
Study to Assess the Efficacy and Safety of Raxone in LHON Patients (LEROS) |
| Creator |
Patrick Yu-Wai-Man, BMedSci, MBBS, PhD, FRCPath, FRCOphth & Nancy J. Newman, MD |
| Affiliation |
(PY) University of Cambridge and Moorfields Eye Hospital, Cambridge and London, UK; (NJN) Emory University School of Medicine, Atlanta, Georgia |
| Subject |
Leber Hereditary Optic Neuropathy; Hereditary Optic Neuropathy; Idebenone; Mitochondrial DNA; Retinal Ganglion Cells |
| Description |
Leber hereditary optic neuropathy (LHON) is a mitochondrial disease resulting in bilateral vision loss. 1,2 Recovery of functional visual acuity (VA) and/or prevention of further VA worsening are desirable outcomes. Idebenone is a soluble short-chain synthetic benzoquinone that helps shuttle electrons directly to complex III, bypassing complexes I and II of the mitochondrial respiratory chain.3,4 This compound is hypothesized to restore mitochondrial function via a number of mechanisms, increasing ATP production and reducing the level of reactive oxygen species.3,4 Idebenone was approved by the European Medicines Agency (EMA) in 2015 for the treatment of LHON at a recommended dose of 300 mg three times/day for patients aged 12 years and older. |
| Date |
2024-03 |
| References |
1. Newman NJ, Yu-Wai-Man P, Biousse V, and Carelli V. Understanding the molecular basis and pathogenesis of hereditary optic neuropathies: towards improved diagnosis and management. Lancet Neurol. 2023; 22:172-188. 2. Carelli V, Carbonelli M, de Coo IF, Kawasaki A, Klopstock T, Lagrèze WA, La Morgia C, Newman NJ, Orssaud C, and Pott JWR, et al. International consensus statement on the clinical and therapeutic management of Leber hereditary optic neuropathym. J Neuro-ophthalmol. 2017; 37:371-381. 3. Haefeli RH, Erb M, Gemperli AC, Robay D, Courdier Fruh I, Anklin C, Dallmann R, and Gueven N. NQO1-dependent redox cycling of idebenone: effects on cellular redox potential and energy levels. PLoS One. 2011; 6: e17963. 4. Gueven N, and Faldu D. Idebenone treatment in Leber's hereditary optic neuropathy: rationale and efficacy. Expert Opin Orphan Drugs. 2013; 1:331-339. 5. Klopstock T, Yu-Wai-Man P, Dimitriadis K, Rouleau J, Heck S, Bailie M, Atawan A, Chattopadhyay S, Schubert M, and Garip A, et al. A randomized placebo-controlled trial of idebenone in Leber's hereditary optic neuropathy. Brain. 2011; 134;2677-2686. |
| Language |
eng |
| Format |
video/mp4 |
| Type |
Image/MovingImage |
| Source |
2024 North American Neuro-Ophthalmology Society Annual Meeting |
| Relation is Part of |
NANOS Annual Meeting 2024: Global Perspectives: Lost in translation? How major neuro-ophthalmic trials are applied locally (US/Canada) versus internationally |
| Collection |
Neuro-Ophthalmology Virtual Education Library: NANOS Annual Meeting Collection: https://novel.utah.edu/collection/nanos-annual-meeting-collection/ |
| Publisher |
North American Neuro-Ophthalmology Society |
| Holding Institution |
Spencer S. Eccles Health Sciences Library, University of Utah |
| Rights Management |
Copyright 2024. For further information regarding the rights to this collection, please visit: https://NOVEL.utah.edu/about/copyright |
| ARK |
ark:/87278/s6etdjxp |
| Setname |
ehsl_novel_nam |
| ID |
2589929 |
| Reference URL |
https://collections.lib.utah.edu/ark:/87278/s6etdjxp |
