Study Identifies Potential New Avenue for Treating Pompe Disease

< p > Contact: Amara Omeokwe < br / > Phone: [phone] < br / > Email:   < a href= " mailto:[e-mail] " > [e-mail] < /a > < br / > < a href= " https://www.dukemedicine.org/ " > https://www.dukehealth.org < /a > < /p > < p > FOR IMMEDIATE RELEASE on Monday, July 25, 2016 < /p > < p > < span style= " line-height: 1.2em; " > DURHAM, N.C. -- Researchers at Duke Health have identified a potential new avenue for treating Pompe disease, a rare condition caused by the build-up of glycogen, a storage form of sugar, in cardiac and skeletal muscle, the liver and other tissues, due to deficiency of a particular enzyme.   < /span > < /p > < p > There are more than 13 diseases like Pompe, which are known as glycogen storage diseases (GSDs), and they cause potentially fatal damage to the liver, heart and skeletal muscles. Pompe disease is treated mainly through enzyme replacement therapy, which helps prevent glycogen accumulation in lysosomes, membrane-enclosed structures found within animal cells. The basic research and clinical translation behind that lifesaving therapy was conducted at Duke. < /p > < p > The current study, co-led by < a href= " https://pediatrics.duke.edu/faculty/priya-sunil-kishnani-mbbs " > Dr. Priya S. Kishnani < /a > , M.D., professor and chief in the Division of Medical Genetics at Duke University School of Medicine, and < a href= " https://pediatrics.duke.edu/faculty/baodong-sun-phd " > Baodong Sun < /a > , Ph.D., associate professor...

Source: DukeHealth.org: Duke Health Features - July 26, 2016 Category: Pediatrics Tags: Duke Medicine Source Type: news