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Ubidecarenone in the treatment of mitochondrial myopathies: a multi-center double-blind trial |
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| Authors: | N Bresolin C Doriguzzi C Ponzetto C Angelini I Moroni E Castelli E Cossutta A Binda A Gallanti S Gabellini G Piccolo A Martinuzzi E Ciafaloni E Arnaudo L Liciardello ACarenzi and G Scarlato |
| Institution: | aInstitute of Clinical Neurology, University of Milan, Milan (Italy) bInstitute of Clinical Neurology, University of Torino, Turin (Italy) cInstitute of Clinical Neurology, University of Padua, Padua (Italy) dDepartment of Biomedical Sciences and Oncology, University of Torino, Turin (Italy) eInstitute of Clinical Neurology, University of Bologna, Bologna (Italy) fInstitute of Clinical Neurology, University of Pavia, Pavia (Italy) gResearch Laboratory, Zambon Pharmaceutical Company, Bresso Milano (Italy) hDepartment of Cardiology, Ospedale Maggiore, Policlinico, Milan (Italy) |
| Abstract: | Forty-four patients with mitochondrial myopathies were treated with Ubidecarenone (CoQ10) for 6 months in an open multi-center trial. No side effects of the drug were observed. Sixteen patients showing at least 25% decrease of post-exercise lactate levels were selected as responders. Responsiveness was apparently not related to CoQ10 level in serum and platelets or to the presence or absence of mtDNA deletions. The responders were treated for a further 3 months with CoQ10 or placebo in the second blind part of the trial; no significant differences were observed between the 2 groups. It is not clear why CoQ10 had therapeutic effects in some patients and not in others with the same clinical presentation and biochemical defect, and we failed to identify candidate responders before treatment. At the dose of CoQ10 used in this study (2 mg/kg/day) the therapy requires a long administration time before a response is seen. |
| Keywords: | Mitochondrial myopathy Ubiquinone mtDNA deletion |
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