Priya S. Kishnani is the Chen Family Distinguished Professor of Pediatrics, chief of the Division of Medical Genetics, a professor in the Department of Molecular Genetics and Microbiology, core faculty member of the Duke Innovation and Entrepreneurship Initiatives, and member of the Duke Clinical Research Institute.
Throughout her career, Kishnani has conducted groundbreaking basic and clinical research in the fields of glycogen storage disease genetics and therapeutics. She has demonstrated sustained achievements and leadership in research at Duke University School of Medicine in her role as an international principal investigator and Food and Drug Administration (FDA) liaison in pivotal clinical trials, where she was critical in study design, development of endpoints, and identification of risk factors. Kishnani’s landmark discoveries led to the advent of enzyme replacement therapy in Pompe disease. She was able to take the lessons learned from the bench and translate them to the bedside. As the lead principal investigator, she designed and conducted multicenter and multinational clinical trials of Myozyme, which resulted in FDA and European Medicines Agency's approval of the first-ever therapy for Pompe disease.
Kishnani developed the immune tolerance induction protocol for the prevention of the development of antibody responses and management of entrenched antibody response to therapeutic agents, which led to an FDA label change. In addition to her work in glycogen storage diseases, she is a pioneer in the development of novel approaches to improve cognitive function in Down syndrome. She is the Director of the Duke Health Rare Disease Center which was designated a Rare Disease Center of Excellence for research and patient care by the National Organization for Rare Disorders. Duke is internationally recognized in many rare diseases, particularly as a leader in the fields of glycogen storage diseases and lysosomal storage diseases.
Kishnani has lectured throughout the world, served on executive committees of the National Institute of Child Health and Human Development, the North Carolina Genetics Advisory Committee, the National Glycogen Storage Disease Association, and the Duke Department of Pediatrics, and received awards for research, mentorship, teaching, and clinical expertise.
Education: University of Bombay; Jai Hind College, TN Medical College, Bombay
Training: Duke University School of Medicine
Current Title: Chen Family Distinguished Professor of Pediatrics, Duke University School of Medicine
Updated April 22, 2022