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Researchers at UPMC Children’s Hospital of Pittsburgh have taken on multiple challenges in gastroenterology disorders. Pancreatitis, liver disease, biliary atresia, and inflammatory bowel disease are just a few of the many areas of focus.
With funding from the National Institutes of Health, Children’s gastroenterology laboratories provide leadership in: understanding the physiology of lipases – enzymes that digest fats – and, more specifically, the roles these proteins play in digestion and disease; characterizing the function and regulation of intestinal transporters for water and bile acids; and the pathophysiology and treatment of alpha-1-antitrypsin deficiency, the most common genetic disease of the liver in childhood.
Children’s gastroenterologists also head multiple clinical studies. Liver disease is under assault from Children’s pediatric gastroenterologists working to advance potential therapies that range from increasing our understanding of biliary atresia and acute liver failure, liver diseases often treated with liver transplantation, to transplanting hepatocytes as a possible cure for many childhood liver diseases. Clinical studies in pediatric inflammatory bowel disease are also a focus of Children’s gastroenterology researchers. These studies include participation in local and national registries to gather data about outcome and treatment of children with inflammatory bowel disease and an innovative and novel study to define the role of behavioral therapy in treating children with inflammatory bowel disease.
Long-term Safety and Durability of the Therapeutic Effect of LUM001, an Apical Sodium-dependent Bile Acid Transporter Inhibitor (ASBTI), in the Treatment of Cholestatic Liver Disease in Pediatric Subjects with Alagille Syndrome
A Preliminary Study of the Efficacy and Safety of Carbamazepine in Severe Liver Disease Due to Alpha-1-Antitrypsin Deficiency
Biliary Atresia Study in Infants, Children and Adults
A Long-Term Safety Study of Maralixibat, an Apical Sodium-Dependent Bile Acid Transporter Inhibitor, in the Treatment of Cholestatic Liver Disease in Subjects Who Previously Participated in a Maralixibat Study
Longitudinal Study of Genetic Causes of Intrahepatic Cholestasis (LOGIC)
A Prospective Database of Infants with Cholestasis
Sex Differences in Statural Growth Impairment in Pediatric Crohn’s Disease
Treating Disrupted Sleep in Individuals with Inflammatory Bowel Disease: A Novel Adjunctive Therapy for Chronic Inflammatory Illness
Study on the Genetic, Environmental and Microbial Interactions that Cause IBD (GEM Project)
Autologous Stem Cell Transplantation with CD34 Selected Peripheral Blood Stem Cells (PBSC) in Pediatric and Young Adult Patients with Severe Crohn's Disease
Study to Assess the Combination Regimen of Elbasvir/Grazoprevir in Pediatric Participants With Chronic Hepatitis C Infection
An Open Label Multicenter Study to Evaluate the Pharmacokinetics, Safety, and Efficacy of Glecaprevir/Pibrentasvir in Pediatric Subjects with Genotypes 1-6 Chronic Hepatitis C Virus Infection
Using Patient Data to Transform Care and Improve Outcomes for Children, Adolescents and Young Adults with Inflammatory Bowel Disease
Intestinal Care and Rehabilitation Center Database
FibroScan® in Pediatric Cholestatic Liver Disease Study
Longitudinal Study of Mitochondrial Hepatopathies
A 5-year Longitudinal Observational Study of Patients with Nonalcoholic Fatty Liver or Nonalcoholic Steatohepatitis
Pediatric Longitudinal Cohort Study of Chronic Pancreatitis (INSPPIRE 2)
MRX-502: Randomized Double-blind Placebo-controlled Study to Evaluate the Efficacy and Safety of Maralixibat in the Treatment of Subjects with Progressive Familial Intrahepatic Cholestasis
MRX-503: An Extension Study to Evaluate the Long-term Safety and Efficacy of Maralixibat in the Treatment of Subjects with Progressive Familial Intrahepatic Cholestasis
Compassionate Use of Omegaven in the Treatment of Parenteral Nutrition-Associated Hepatic Injury
Arjumand Ghazi, PhD
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One Children’s Hospital Way
4401 Penn Ave.
Pittsburgh, PA 15224
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