Chronic pediatric liver disease is a devastating group of conditions that have a profound effect on children, their families, and our society. Biliary atresia and the other cholestatic liver diseases studied by this network account for over half of liver transplants performed in children in the United States. A better understanding of the diseases studied by this network will help provide better care of patients with chronic liver disease. The gaps in our current knowledge regarding these diseases provide compelling scientific justification for the continuation of ChiLDReN. As stated in the RFA, the primary goal of this proposal is to continue clinical and translational research on rare pediatric liver disease that include: biliary atresia;Alagille syndrome, alpha-1-antitrypsin deficiency, progressive familial intrahepatic cholestasis syndromes, bile acid synthesis defects, mitochondrial hepatopathies, idiopathic neonatal hepatitis, and cystic fibrosis liver disease. It is anticipated that the network will consist of up to 15 clinical sites and a single data coordinating center (DCC). The DCC for the ChiLDReN study is charged with providing coordination, communications and logistical support, clinical study design, centralized data management, biosample management, quality assurance, and analytical support to the research sites and the NIDDK Project Scientist for all ChiLDReN studies. The proposing team is submitting this response to RFA-DK-13-011 to continue as the DCC for the network.
The Childhood Liver Disease Research Network (ChiLDReN)* studies babies, children, and young adults who have rare mostly cholestatic liver diseases. The researchers will ask patients to participate in studies that will help us: 1) Develop new tests to better diagnose these diseases, 2) Learn more about what causes these diseases, 3) Develop better treatment options for children living with these diseases, 4) Develop better treatment options for children who receive a liver transplant for these diseases *When referencing the current research network, we use the acronym ChiLDREN. When referencing the proposed network described in RFA-DK-13-011: Continuation of ChiLDReN, we use the acronym ChiLDReN.
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