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Closed Access item Coenzyme Q10-responsive ataxia: 2-year-treatment follow-up

Authors:Pineda, Mercé
Aracil, Asunción
O'Callaghan, Mar M.
Más, Ana
Espinos, Carmen
Martínez-Rubio, Dolores
Palau Martínez, Francesc
Navas, Plácido
Briones, Paz
Artuch, Rafael
Keywords:Coenzyme Q10 deficiency, Mitochondrial disorders, Ataxia, Cerebellum, Pediatric patients
Issue Date:13-Apr-2010
Publisher:John Wiley & Sons
Citation:Movement Disorders 25(9): 1262-1268 (2010)
Abstract:We assessed the clinical outcome after coenzyme Q10 (CoQ10) therapy in 14 patients presenting ataxia classified into two groups according to CoQ10 values in muscle (deficient or not). We performed an open-label prospective study: patients were evaluated clinically (international cooperative ataxia rating scale [ICARS] scale, MRI, and videotape registration) at baseline and every 6 months during a period of 2 years after CoQ10 treatment (30 mg/kg/day). Patients with CoQ10 deficiency showed a statistically significant reduction of ICARS scores (Wilcoxon test: P = 0.018) after 2 years of CoQ10 treatment when compared with baseline conditions. In patients without CoQ10 deficiency, no statistically significant differences were observed in total ICARS scores after therapy, although 1 patient from this group showed a remarkable clinical amelioration. Biochemical diagnosis of CoQ10 deficiency was a useful tool for the selection of patients who are good candidates for treatment as all of them responded to therapy. However, the remarkable clinical response in 1 case without CoQ10 deficiency highlights the importance of treatment trials for identification of patients with CoQ10-responsive ataxia.
Description:7 páginas, 2 figuras, 2 tablas.
Publisher version (URL):http://dx.doi.org/10.1002/mds.23129
E-ISSNmetadata.dc.identifier.doi = DOI:1531-8257
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