Abstract of Dr. Priya Kishnani Interview

Dr. Kishnani speaks about: her family’s background, particularly her mother’s background as a physician in India; coming to the United States; medical genetics; Duke’s strength in medical genetics; comparisons between medicine in the US and in India; Pompe disease; how she became involved with children with Pompe disease; Dr. YT Chen; educating the medical establishment that children with Pompe disease could be treated; successes of the treatment; details of the treatment; setting up the clinical trials for Pompe disease; intensive nature of setting up the clinical trials for Pompe disease; commitment level of coordinating these trials; physicians in other countries giving treatment for Pompe disease locally; working with Genzyme Corporation to develop the drug used in treatment of Pompe disease; developing an appropriate relationship with industry when working in academic medicine; translational medicine; her work in trials with children with Down syndrome; treatment versus cure for Pompe disease and Down syndrome; center for treatment of rare diseases at Duke; special considerations for clinical trials for treatment of rare diseases; her continuing commitment to work with Pompe disease; being a woman in medicine.