Precision medicine in Wolfram syndrome

Our current focus is to delay the progression of Wolfram Syndrome using a drug already approved for other conditions. We have three goals, Raise Awareness, Improve Clinical Care, and Provide a Cure, and this is the first step for our third and the most important goal, Provide a Cure.

One of the things we need to start immediately is to completely dissect out the molecular wiring in brain cells and pancreatic beta cells in patients with Wolfram syndrome. This strategy has been tested and proven effective in developing novel therapies for cancer patients. This is aligned with the Precision Medicine Initiative led by Dr. Francis Collins. We would like to sequence entire genome of our patients and create a map of signaling pathways altered in Wolfram syndrome patients. With the information obtained from these studies, we will design personalized therapies.

Thank you so much for your continued support and encouragement. I am grateful.

Kindest regards,

Fumi Urano