Cincinnati (Ohio), April 16 -- : University of Cincinnati researchers have found a newer, more effective treatment for the disease Pompe that could become the new standard of care for the rare condition.
Rare diseases are sometimes the most difficult to treat because of a lack of research and fewer participants to study. An example would be those who have Pompe disease, a genetic condition when a body can't make a protein that breaks down a complex sugar, called glycogen, for energy. Too much glycogen builds up and damages muscles and organs. The disease causes muscle weakness and trouble breathing and can affect the heart and muscles.
Hani Kushlaf, MD, an associate professor in both the Department of Neurology and Rehabilitation Medicine...