The purpose of this research study is to develop a new rating scale to be used in clinical trials (drug or intervention trials) in mitochondrial disease. The aim of the rating scale is to measure symptom changes over short periods of time to determine the efficacy of a drug or other intervention (eg exercise). IRB 16-013364
Mitochondrial Medicine Clinical Studies
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We are studying an investigational drug called sodium dichloroacetate (DCA). Investigational means that the study drug has not been approved by the US Food and Drug Administration (FDA) and that its use in this study is experimental. This a purpose of this research study is to determine whether DCA is a helpful treatment for patient with pyruvate dehydrogenase complex (PDC) deficiency (PDCD).