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Clinical Studies and Research

Searching for new ways to treat genetic conditions.

Children’s Hospital’s Brain Care Institute, in collaboration with the University of Pittsburgh, is committed to the research of mitochondrial and metabolic disorders. In addition to studying the fundamental issues underlying these disorders, we’re developing better therapeutic approaches and advanced treatments, and uncovering more clinical implications of mutations in genes. Our active clinical research program works with other genetic programs worldwide to develop studies and clinical trials, including:

  • Phase 2 Trial of Triheptanoin for treatment of Long Chain Fatty Acid Oxidation Disorders
  • A Longitudinal Study of Mitochondrial Hepatopathies, which is a study of rare liver diseases that occur in infants, children, and young adults
  • The Development of Chemical Chaperone for Medium Chain Acyl-CoA Dehydrogenase Deficiency, which studies methods of treating an inborn error of fatty acid metabolism
  • A Diabetes Vaccine Study that explores new ways to suppress Type 1 diabetes

Learn more about mitochondrial and metabolic research and clinical studies.

Last Update
September 26, 2012
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Last Update
September 26, 2012