title: Is Leber hereditary optic neuropathy treatable? Encouraging results with idebenone in both prospective and retrospective trials and an illustrative case.
abstract: A 31-year-old woman developed subacute bilateral visual loss over a 2-week period. Two months later, the diagnosis of Leber hereditary optic neuropathy (LHON) 11778/ND4 was established and the patient was treated with 900 mg of idebenone daily. Over the ensuing 9 months, visual acuity improved from 20/200 to 20/25 in each eye with near-total resolution in visual field abnormalities. Our case report is in agreement with 2 large published series of patients with LHON treated with idebenone, raising hope for treatment of this visually devastating mitochondrial disorder.
authors.affiliation: Department of Ophthalmology, Doheny Eye Institute, Keck School of Medicine, University of Southern California, Los Angeles, CA 90033, USA.
authors.forename: Esfandiar J
authors.initials: EJ
authors.lastname: Sabet-Peyman
authors.forename: Khizer R
authors.initials: KR
authors.lastname: Khaderi
authors.forename: Alfredo A
authors.initials: AA
authors.lastname: Sadun
mesh_terms: D000328:Adult; D000975:Antioxidants / Q000008:administration & dosage*; D002986:Clinical Trials as Topic; D005260:Female; D006801:Humans; D029242:Optic Atrophy, Hereditary, Leber / Q000188:drug therapy* / Q000503:physiopathology; D011446:Prospective Studies; D020127:Recovery of Function / Q000187:drug effects*; D012189:Retrospective Studies; D016896:Treatment Outcome; D014451:Ubiquinone / Q000008:administration & dosage / Q000031:analogs & derivatives*
chemical_list: D000975:Antioxidants; D014451:Ubiquinone; C036619:idebenone