Dr. Priya Kishnani was born in Bombay, India. She received her MD in Bombay in 1990. She came to the United States in 1991 and became a resident at Duke University Medical Center. At Duke, Dr. Kishnani became involved in designing the first clinical trials for a new treatment of Pompe disease, a rare disease affecting the heart and the muscles. The treatment, using a drug called Myozyme, was ultimately successful, and it achieved FDA approval for treatment of the disease in 2006. Dr. Kishnani has dedicated her career to advancing the treatment of Pompe disease globally. She has become an expert in designing clinical trials for treatments of rare diseases, and is often sought as a consultant for this expertise. She is published widely.