THE UREA CYCLE DISORDERS CONSORTIUM: SETTING UP A MULTI-INSTITUTIONAL RESEARCH
NETWORK IN RARE DISEASES
Batshaw ML
Children’s National Medical Center, Washington, DC
The Urea Cycle Disorders (UCD) Consortium was established in 2003, funded through a grant from the
Office of Rare Diseases Research and the National Institute of Child Health and Human Development of
the NIH as one of the initial 10 Rare Diseases Clinical Research Centers. Starting with 5 sites in the U.S.,
it has evolved into an international consortium with 11 sites in the U.S., one in Canada and two in
Europe. The additional sites have been funded primarily by philanthropy. The UCDC has a four-fold
mission: 1) to perform collaborative clinical research including longitudinal studies of individuals with
urea cycle disorders and clinical trials; 2) to train clinical investigators in rare diseases; 3) to perform
pilot/demonstration clinical research projects; and 4) to provide access on a website to information
related to UCD for basic and clinical researchers, academic and practicing physicians, patients and the
lay public. This talk will describe lessons learned from developing this multi-institutional network and
discuss some of the results that have come from these studies.