Old drug offers new hope against rare, deadly childhood disease
Tuesday, April 2, 2013 - 16:30
in Health & Medicine
Washington University School of Medicine in St. Louis is playing a leading role in one of the National Institutes of Health’s (NIH) first clinical trials to improve treatments for rare and neglected diseases. In this case, the disease is Niemann-Pick Type C, a disorder that causes excess cholesterol to accumulate in the brain, liver and spleen. It affects about 500 children worldwide, leads to neurodegeneration, and usually causes death in the first two decades of life. The compound to be tested is called cyclodextrin, a cyclic sugar long used as an ingredient in other drugs.