Pioneering Research and Treatment for Pompe Disease | Duke Health

Duke Health geneticist Priya Kishnani, M.D., and patient Haley Hayes and her mother Krystal Hayes describe advancements in research and treatment of Pompe disease at Duke University, including support for newborn screening for rare diseases. The only FDA-approved treatment for Pompe disease -- enzyme replacement therapy -- was developed by genetic specialists at Duke.

FULL STORY: http://ow.ly/SLtS30jcEQk