Why a Common Diabetes Drug May Be Able to Treat a Rare Disease

A widely used drug to treat diabetes may hold promise as a therapeutic for patients with Maple Syrup Urine Disease, a rare, inherited metabolic disorder involving the dysfunction of an enzyme needed to break down three essential amino acids. The findings, which appear in a new study in Nature Scientific Reports come from researchers at the Buck Institute for Research on Aging. We spoke to Arvind Ramanathan, senior author of the study and a member of the Buck faculty, about the study, what it says about the ability to understand more common diseases through the study of rare diseases, and how repurposing existing therapies holds promise of brining new treatments to rare disease patients who are without them.