Results for Natural History

Using translational approaches that encompass genomic studies, biomarker development, disease modeling, natural history studies, and clinical trials, Dr. Vanderver seeks to improve the quality of life and lifespan of individuals living with leukodystrophies or heritable disorder of myelin. She leads the Leukodystrophy Center of Excellence at Children's Hospital of Philadelphia.

Dr. Young’s translational research program aims to understand mechanisms underlying interstitial and rare lung diseases and develop new strategies to treat these disorders. Her laboratory focuses on the roles of epithelial cells in alveolar homeostasis, injury, and repair.

Dr. Muir investigates the mechanisms underlying esophageal fibrosis to improve therapeutic and diagnostic approaches.

Dr. Loomes' research is focused on clinical and translational studies in pediatric liver disease. She works with National Institutes of Health-funded national consortia to conduct studies investigating the etiology and treatment for rare pediatric liver diseases including biliary atresia, Alagille syndrome, and others. Dr. Loomes also collaborates with other investigators at Children’s Hospital of Philadelphia to investigate genetic causes of pediatric liver disease.

Dr. Silverman studies the fundamental aspects of early-life commensal microbes that influence immune system development and function. He discovered that the MHC-II E molecule prevents type 1 diabetes by shaping the intestinal microbiota early in life.

Dr. Hartung's clinical and translational research program focuses on autosomal recessive polycystic kidney disease and other genetic kidney diseases, development of new kidney and liver imaging biomarkers, and neurocognitive outcomes in children with chronic kidney disease.

Dr. Lerman's research interests include non-infectious uveitis and temporomandibular (TMJ) arthritis. Her studies examine the utility of biologic agents to achieve, and maintain, uveitis control. Up to 80 percent of children with juvenile idiopathic arthritis (JIA) have potentially erosive TMJ arthritis, and she is exploring ways to best identify and monitor this often asymptomatic manifestation of JIA.

As a bioengineer, Dr. Arbogast's research focuses on pediatric injury biomechanics, injury causation and the effectiveness of safety products for children with a concentration in the safety of children and youth in motor vehicle crashes as well as pediatric concussion.

Dr. Ahrens-Nicklas works to understand why patients with inherited biochemical disorders often suffer severe, untreatable neurologic and cardiac symptoms. She strives to elucidate the link between biochemistry and network excitability, in order to drive new approaches to therapy.

Dr. Curry's research focuses on the epidemiology and prevention of motor vehicle crashes in children and adolescents.