title: Efficacy of idebenone for respiratory failure in a patient with Leigh syndrome: a long-term follow-up study.
abstract: Respiratory failure can be the direct cause of death in patients with Leigh syndrome. Unfortunately, no effective treatment strategy is available. Here, we report successful treatment of a patient with Leigh syndrome using idebenone, a derivative of coenzyme Q-10. The patient's brainstem function, especially respiratory function, improved after idebenone treatment. Idebenone may be worth trying in patients with Leigh syndrome.
authors.affiliation: Department of Pediatric Neurology, Takuto Rehabilitation Center for Children, Sendai 982-0241, Japan. khaginoya@silk.ocn.ne.jp
authors.forename: Kazuhiro
authors.initials: K
authors.lastname: Haginoya
authors.forename: Shigeaki
authors.initials: S
authors.lastname: Miyabayashi
authors.forename: Masahiro
authors.initials: M
authors.lastname: Kikuchi
authors.forename: Akira
authors.initials: A
authors.lastname: Kojima
authors.forename: Katsuya
authors.initials: K
authors.lastname: Yamamoto
authors.forename: Kiyoshi
authors.initials: K
authors.lastname: Omura
authors.forename: Mitsugu
authors.initials: M
authors.lastname: Uematsu
authors.forename: Naomi
authors.initials: N
authors.lastname: Hino-Fukuyo
authors.forename: Soichiro
authors.initials: S
authors.lastname: Tanaka
authors.forename: Shigeru
authors.initials: S
authors.lastname: Tsuchiya
mesh_terms: D000293:Adolescent; D001933:Brain Stem / Q000187:drug effects / Q000503:physiopathology; D016057:Evoked Potentials, Auditory, Brain Stem / Q000187:drug effects; D005500:Follow-Up Studies; D006801:Humans; D019344:Lactic Acid / Q000134:cerebrospinal fluid; D007888:Leigh Disease / Q000150:complications*; D008297:Male; D017286:Polysomnography; D012119:Respiration / Q000187:drug effects; D012131:Respiratory Insufficiency / Q000188:drug therapy* / Q000209:etiology; D014451:Ubiquinone / Q000031:analogs & derivatives* / Q000627:therapeutic use
chemical_list: D014451:Ubiquinone; D019344:Lactic Acid; C036619:idebenone