More importantly, we demonstrate that short- and long-acting GLP-1R agonists confer protection in different human models of Wolfram syndrome, both in vitro in clonal and primary human beta cells, Wolfram iPSC-derived beta cells, NPCs and cerebellar neurons, and in vivo in humanised mice grafted with Wolfram iPSC-derived beta cells. This evidence concerns the gene GLP1R and Wolfram syndrome.