GLP-1 agonist treatment significantly improved the glycemic control in one Wolfram syndrome 1 patient, and in a second patient with a dominant form of Wolfram syndrome. A clinical trial with exenatide in Wolfram syndrome 2 patients was launched, and a second one with Liraglutide in Wolfram syndrome 1 patients has been recently announced. This evidence concerns the gene GLP1R and Wolfram syndrome.