Alice and Y.T. Chen Center for Genetics and Genomics
In April 2006, Myozyme (recombinant acid alpha-glucosidase) was approved by the FDA for use in patients with Pompe disease, representing the culmination of efforts by YT Chen, MD, PhD, and colleagues to develop a cure for this deadly condition. With the goal of reproducing the bench-to-bedside approach that generated Myozyme and now offers hope for patients with Pompe disease, the Chens made a gift to the Department of Pediatrics to establish the Alice and YT Chen Pediatric Genetics and Genomics Research Center. This initiative focuses on single gene disorders amenable to enzyme replacement therapy or gene therapy. Priya Kishnani, MD, Chief of the Division of Medical Genetics and the lead investigator in the international clinical trials of Myozyme, serves as the Medical Director of the Center.
Jean and George Brumley, Jr. Neonatal Perinatal Research Institute (NPRI)
The Jean and George Brumley, Jr. Neonatal Perinatal Research Institute (NPRI), established in 1997, is dedicated to developmental biology and focuses its research on the serious health problems of newborns. Among the areas of active investigation are the mechanisms of brain, lung and cardiovascular injury and the cellular etiology of birth defects.
International Pediatric Fungal Network
The multi-center International Pediatric Fungal Network (PFN) was created to gain a complete understanding of the scope and character of pediatric fungal infections in order to improve the care of our patients. The primary mission of the PFN is to increase the knowledge of pediatric invasive fungal infections and discern any undescribed characteristics or outcomes unique to pediatric patients through a coordinated network of scientific investigation.
Federally Sponsored Research Networks
The Department of Pediatrics participates in a number of federally funded clinical research networks. These networks and their principal investigators are listed below.
Faculty in the Department of Pediatrics are involved in a number of global research initiatives facilitated by the Duke Global Health Institute (DGHI)
. The Duke Global Health Institute is committed to reducing health disparities in the local community and in resource-limited regions of the world. Recognizing that many global health problems arise from economic, social, environmental, political and health care inequalities, DGHI brings together interdisciplinary teams to solve complex health problems and to train the next generation of global health scholars.
As the passion for global health grows within the Duke community, we expect the number of Department of Pediatrics faculty who are involved in global health efforts in education, research, policy or service to expand.
Pediatric Trials Network (PTN)
The Pediatric Trials Network is an alliance of clinical research sites located around the United States that are cooperating in the design and conduct of pediatric clinical trials to improve health care for the youngest patients. The PTN is studying the formulation, dosing, efficacy, and safety of drugs, as well as the development of medical devices, used in pediatric patients. Danny Benjamin, MD, the Kiser-Arena Professor of Pediatrics (Infectious Diseases), is the principal investigator for the Pediatric Trials Network, which is centered at Duke.
Duke Translational Medicine Institute (DTMI)
The Duke Translational Medicine Institute (DTMI)
Pediatrics Core was established to specifically address the increasing need for a coherent and integrated method for the conduct of pediatric clinical and translational research projects due to FDA pediatric initiatives and the proliferation of NIH-funded mechanisms for pediatric research.
The mission of the DTMI Pediatrics Core resource is to provide a leading pediatric academic enterprise that promotes measurable improvements in the health of children by fostering the translation of new scientific discoveries into clinical practice through collaborative clinical and translational research. The Pediatrics Core will provide both the training and the opportunity for established and newly-trained investigators to use their skills in pediatric translational studies and clinical research.
The Pediatric Neurocognitive Outcomes unit, supported by the DTMI, offers investigators assistance in assessing various domains of neuropsychological function, behavioral/psychosocial functioning, and quality of life in children.
To learn more about the organizational objectives, major initiatives and faculty affiliated with DTMI Pediatrics, please visit the Child Health Research Core section of the DTMI web site
Duke Clinical Research Institute (DCRI)
The Duke Clinical Research Institute (DCRI)
, the world's largest academic clinical research organization, conducts pediatric clinical trials on an unprecedented scale. These trials involve thousands of participants from across the globe and combine the clinical expertise and academic leadership of a premier teaching hospital with the full-service operational capabilities of a major contract research organization.
DCRI pediatric research experience ranges from pharmacokinetic studies in neonates to large, multi-center trials in adolescent populations. The faculty are renowned, experienced researchers in all of the most crucial therapeutic areas, and are also practicing physicians, observing the results of hard-won evidence right at the bedside.
Added to this academic and clinical expertise is the unique operational capability of the DCRI making it the undisputed world leader in pediatric clinical research.
Duke Clinical Research Unit (DCRU)
The Duke Clinical Research Unit (DCRU)
provides specialized facilities, staff, resources, and expertise in conducting early phase clinical research with the goal of facilitating the understanding of human conditions, translating basic research findings into patient care, and developing new therapies.
The following services are available for early phase pediatric clinical research: