|A prospective study of biochemical parameters reflective of metabolic control in propionic acidemia|
Individuals of any age with propionic acidemia who have not had a liver transplant may be eligible to take part in a research study on biomarkers
being done by Dr. Loren Pena at Duke University Medical Center.
Biomarkers are compounds that can be measured by laboratory tests in body fluids such as blood and urine and are helpful in predicting disease states. For example, cholesterol level is a biomarker for heart disease. For patients with some metabolic disorders, biomarkers can be helpful in guiding treatments (such as the amount of protein a person can eat) and can predict whether a person is at risk to develop a health problem associated with that metabolic disorder. Currently, there is no standard set of laboratory tests recommended to help guide treatment for people with propionic acidemia. This is because little is understood about which biomarkers are most helpful. The goals of this study are:
For more information, please contact:
Jennifer Goldstein, PhD, CGC