Chronic treatment of mitochondrial disease patients with dichloroacetate

BA Barshop, RK Naviaux, KA McGowan… - Molecular genetics and …, 2004 - Elsevier
Clinical features are reported for 37 patients with various mitochondrial disorders, treated
with sodium dichloroacetate (DCA) for 3 weeks to 7 years (mean 3.25 years) at 11–
50mg/kg/day (34.6±13.1) in an open-label format. DCA pharmacokinetics showed half-times
approximately 86min for the first intravenous dose of 50mg/kg, 3.2 h for a subsequent
intravenous dose 4–6h later, and 11h after continued oral dosing of 12.5–25mg/kg twice
daily. Basal blood and CSF lactate (mean values at entry 29.6 and 46.8 mg/dL, respectively) …