Molecular Pathogenesis and Experimental Therapeutics

The Hepatorenal Fibrocystic Disease (HFRD) Translational Core Center was founded in 2005 by Lisa Guay-Woodford, M.D., during her tenure at the University of Alabama at Birmingham and funded through an National Institutes of Health P30 mechanism. Autosomal recessive polycystic kidney disease (ARPKD) and other hepatorenal fibrocystic diseases are relatively rare recessive disorders, but constitute an important set of childhood nephropathies.

Children’s National is one of only four sites nationwide that are part of the Eunice Shriver Kennedy National Institute of Child Health and Development funded Research Centers in Pediatric Developmental Pharmacology.

The Rare Diseases Clinical Research Center (RDCRC) on Urea Cycle Disorders (UCDC), originally funded by the National Institutes of Health in 2003, consists of 15 U.S. and two international sites and involves more than 50 investigators and staff.