Controlled clinical trial of dichloroacetate for treatment of congenital lactic acidosis in children.
Pediatrics. 2006 May;117(5):1519-31
Stacpoole PW et al.
This landmark study is the first to evaluate DCA in a controlled trial for children with mitochondrial disorders. Although there was no improvement in neurological outcome, the drug was proven to be safe. Perhaps earlier administration or administration at higher doses could have improved the outcome.
For more information on mitochondrial disorders, see part 10 of OMMBID.
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Philippe Campeau, MD
Resident in Medical Genetics at McGill University
OMMBID Blog Administrator