RARECast: A Common Link in a Group of Rare Diseases Creates an Opportunity for Repurposing

A group of rare, genetic, and metabolic diseases known as lysosomal storage disorders are largely without treatments. Mark Noble believes one way of accelerating the development of therapies for this group of disorders is to look at them collectively instead of individually, and see whether existing drugs might serve as treatments. Noble, Professor of Genetics and of Neurobiology and Anatomy at the University of Rochester Medical Center, recently co-authored a study in PLoS Biology (bit.ly/2kPbUzJ) that suggests repurposed drugs could address a common aspect of these diseases and provide therapeutic benefit. We spoke to Noble about his research, the findings of this common link in this group of diseases, and the potential implications for using already approved drugs to bring needed treatments to patients.