Inside Duke Medicine
Duke research inspires Hollywood film
posted January 18th, 2010Duke University Medical Center plays a central role in the story behind the story of the new film Extraordinary Measures.
Twenty years ago, Duke pediatric geneticist Y.T. Chen, M.D., Ph.D., began work on the first and only life-saving treatment for a rare metabolic disorder called Pompe disease. Duke pediatric geneticist Priya Kishnani, M.D., is the world’s leading expert on Pompe and was the lead investigator of the trials that would take Chen’s work out of the lab and into humans.
Based on Duke’s research, the FDA in 2006 approved the use of Myozyme, which is manufactured and marketed by Genzyme Corporation. As a result, children living with Pompe worldwide were treated with Myozyme and given their first fighting chance at life.
Duke researchers also helped to develop a test to allow diagnosis within 24 hours.
Extraordinary Measures, which debuts on Jan. 22, tells the story of one man’s quest to obtain treatment for his children.