Cyclodextrin therapy

Patent Granted In 2009, research from the lab of Michael S. Brown and Joseph L. Goldstein, Nobel Prize–winning scientists who pioneered the study of cholesterol metabolism, was published showing how cyclodextrin assists in moving cholesterol out of lysosomes in Niemann-Pick type C disease. Niemann-Pick disease, type C is a rare and fatal lysosomal storage disease causing progressive deterioration of the nervous system and dementia. The inherited genetic condition typically affects young children by interfering with their ability to metabolize cholesterol at the cellular level and life expectancy often does not exceed an individual’s teenage years.