It was a little over 2 years ago when I embarked on this journey with Wolfram syndrome. Last October, I received a devastating blow, one that would change my life and others forever. My youngest daughter, Raquel, was diagnosed with Wolfram Syndrome, a terminal form of diabetes. She had been diagnosed with Type 1 diabetes […]
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.