As I mentioned a few days ago, I could identify several disorders related to Wolfram syndrome. These patients do not develop all the typical symptoms of Wolfram syndrome. They only develop diabetes, deafness, or optic nerve atrophy. They sometimes have mutant Wolfram syndrome genes, but in many cases they don’t have mutant WFS1 genes. They still have something in common, altered endoplasmic reticulum homeostasis.
My big idea is to diagnose these patients using cutting-edge gene-based diagnostics and treat them using similar drugs. I have a few drugs in my mind, and, of course, I need to get approvals from regulatory agencies. I am working on this. My top priority is still to repurpose a drug and start a clinical trial for Wolfram syndrome.
Take care,
Fumi