title: [The use of noben for correction of mitochondrial disorders in Friedrich's disease].
abstract: The results of treatment with noben (idebenon), an improved structural analogue of coenzyme Q10, of 34 patients with Friedrich's disease are presented. In all cases, the clinical diagnosis was confirmed by the presence of a typical mutation, an expansion of trinucleotide GAA-repeats, in the FRDA gene. All patients received noben as a main drug in dosage 5 mg/kg daily during 3 months. An examination of the patients included modern laboratory and instrumental methods, analysis of levels of lactic and pyruvic acids and their ratio in the peripheral blood. Also parameters of lipid peroxidation and mitochondrial dehydrogenase activity in peripheral lymphocytes were studied. Positive changes were found in the majority of patients for muscle strength in extremities, tolerability to physical loadings, general fatigue, movement activity, speech and coordination functions, along with significant improvement of biochemical and cytochemical status. The results obtained suggest a positive effect of noben on cell energy metabolism in this severe disorder from the group of mitochondrial cytopathies.
authors: M V
authors: MV
authors: Ershova
authors: S N
authors: SN
authors: Illarioshkin
authors: V S
authors: VS
authors: Sukhorukov
mesh_terms: D000293:Adolescent; D000328:Adult; D000975:Antioxidants / Q000008:administration & dosage / Q000627:therapeutic use*; D002648:Child; D004252:DNA Mutational Analysis; D004272:DNA, Mitochondrial / Q000235:genetics; D004305:Dose-Response Relationship, Drug; D004734:Energy Metabolism / Q000187:drug effects; D005621:Friedreich Ataxia / Q000175:diagnosis / Q000188:drug therapy* / Q000235:genetics; D006801:Humans; D033862:Iron-Binding Proteins / Q000235:genetics; D015227:Lipid Peroxidation / Q000187:drug effects; D008214:Lymphocytes / Q000201:enzymology; D008875:Middle Aged; D008928:Mitochondria / Q000201:enzymology; D009154:Mutation; D010088:Oxidoreductases / Q000378:metabolism; D016896:Treatment Outcome; D014451:Ubiquinone / Q000008:administration & dosage / Q000031:analogs & derivatives* / Q000627:therapeutic use; D000096923:Frataxin
chemical_list: D000975:Antioxidants; D004272:DNA, Mitochondrial; D033862:Iron-Binding Proteins; D010088:Oxidoreductases; D014451:Ubiquinone; D000096923:Frataxin; C036619:idebenone