Trial of Dichloroacetate in Pyruvate Dehydrogenase Complex Deficiency: (DCA/PDCD)

Clinical Trial ID NCT02616484

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Top papers

Rank Title Journal Year PubWeight™‹?›
1 Guidance for industry: patient-reported outcome measures: use in medical product development to support labeling claims: draft guidance. Health Qual Life Outcomes 2006 26.32
2 Mcl-1; the molecular regulation of protein function. FEBS Lett 2010 2.71
3 Pinning down proline-directed phosphorylation signaling. Trends Cell Biol 2002 2.17
4 Controlled clinical trial of dichloroacetate for treatment of congenital lactic acidosis in children. Pediatrics 2006 2.13
5 Phosphorylation at serine 10, a major phosphorylation site of p27(Kip1), increases its protein stability. J Biol Chem 2000 1.68
6 Phosphorylation stabilizes Nanog by promoting its interaction with Pin1. Proc Natl Acad Sci U S A 2010 1.57
7 Evaluation of long-term treatment of children with congenital lactic acidosis with dichloroacetate. Pediatrics 2008 1.44
8 Interpreting patient-reported outcome results: US FDA guidance and emerging methods. Expert Rev Pharmacoecon Outcomes Res 2011 1.43
9 A controlled clinical trial of dichloroacetate for treatment of lactic acidosis in adults. The Dichloroacetate-Lactic Acidosis Study Group. N Engl J Med 1992 1.42
10 Therapeutic potential of dichloroacetate for pyruvate dehydrogenase complex deficiency. Mitochondrion 2006 1.23
11 The spectrum of pyruvate dehydrogenase complex deficiency: clinical, biochemical and genetic features in 371 patients. Mol Genet Metab 2012 1.22
12 Age-dependent kinetics and metabolism of dichloroacetate: possible relevance to toxicity. J Pharmacol Exp Ther 2007 1.20
13 EPI-743 reverses the progression of the pediatric mitochondrial disease--genetically defined Leigh Syndrome. Mol Genet Metab 2012 1.17
14 The dichloroacetate dilemma: environmental hazard versus therapeutic goldmine--both or neither? Environ Health Perspect 2010 1.16
15 Reversible protein phosphorylation regulates circadian rhythms. Cold Spring Harb Symp Quant Biol 2007 1.15
16 Phase 1 trial of dichloroacetate (DCA) in adults with recurrent malignant brain tumors. Invest New Drugs 2013 1.07
17 Human polymorphisms in the glutathione transferase zeta 1/maleylacetoacetate isomerase gene influence the toxicokinetics of dichloroacetate. J Clin Pharmacol 2011 1.03
18 Phenylbutyrate therapy for pyruvate dehydrogenase complex deficiency and lactic acidosis. Sci Transl Med 2013 1.02
19 Spectrum of neurological and survival outcomes in pyruvate dehydrogenase complex (PDC) deficiency: lack of correlation with genotype. Mol Genet Metab 2012 1.01
20 Long-term safety of dichloroacetate in congenital lactic acidosis. Mol Genet Metab 2013 0.93
21 Dichloroacetate therapy attenuates the blood lactate response to submaximal exercise in patients with defects in mitochondrial energy metabolism. J Clin Endocrinol Metab 2004 0.92
22 Prolonged hypolactatemia and increased total pyruvate dehydrogenase activity by dichloroacetate. Biochem Pharmacol 1982 0.91
23 Phosphoproteomics. Wiley Interdiscip Rev Syst Biol Med 2010 0.89
24 A combined therapeutic approach for pyruvate dehydrogenase deficiency using self-complementary adeno-associated virus serotype-specific vectors and dichloroacetate. Mol Genet Metab 2008 0.89
25 Unified gas chromatographic-mass spectrometric method for quantitating tyrosine metabolites in urine and plasma. J Chromatogr B Analyt Technol Biomed Life Sci 2004 0.89
26 Haplotype variations in glutathione transferase zeta 1 influence the kinetics and dynamics of chronic dichloroacetate in children. J Clin Pharmacol 2014 0.87
27 The spectrum of pyruvate oxidation defects in the diagnosis of mitochondrial disorders. J Inherit Metab Dis 2014 0.87
28 Design and implementation of the first randomized controlled trial of coenzyme CoQ₁₀ in children with primary mitochondrial diseases. Mitochondrion 2012 0.86
29 Caveats when considering ketogenic diets for the treatment of pyruvate dehydrogenase complex deficiency. J Pediatr 2001 0.86
30 Liquid chromatography-tandem mass spectrometry method for the simultaneous determination of delta-ALA, tyrosine and creatinine in biological fluids. Clin Chim Acta 2004 0.85
31 Determination of dichloroacetate and its metabolites in human plasma by gas chromatography-mass spectrometry. J Chromatogr B Biomed Sci Appl 1997 0.81
32 Preparation and stability of intravenous solutions of sodium dichloroacetate (DCA). J Parenter Sci Technol 1992 0.77
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