Wolfram syndrome is a rare genetic spectrum disorder characterized by insulin-dependent
diabetes mellitus, optic nerve atrophy, and progressive neurodegeneration, and ranges from
mild to severe clinical symptoms. There is currently no treatment to delay, halt, or reverse the
progression of Wolfram syndrome, raising the urgency for innovative therapeutics for this
disease. Here, we summarize our vision for developing novel treatment strategies and
achieving a cure for Wolfram-syndrome-spectrum disorder.