Using a quantitative high-throughput screening strategy, researchers at the National Institutes of Health (NIH) have identified three new classes of small molecules that may prove useful for treating Gaucher disease, an inherited disorder that disrupts a cell’s ability to break down and dispose of certain cellular waste products. The findings, reported in the online edition of the Proceedings of the National Academy of Sciences during the week of July 23-27, could lead to a new therapeutic approach in which a defective enzyme is corrected by an easy-to-take oral medication.

Using a quantitative high-throughput screening strategy, researchers at the National Institutes of Health (NIH) have identified three new classes of small molecules that may prove useful for treating Gaucher disease, an inherited disorder that disrupts a cell’s ability to break down and dispose of certain cellular waste products. The findings, reported in the online edition of the Proceedings of the National Academy of Sciences during the week of July 23-27, could lead to a new therapeutic approach in which a defective enzyme is corrected by an easy-to-take oral medication.