We have a few candidate drugs for delaying the progression of Wolfram syndrome. What needs to be done to move forward? Here are my plans.
1. Dose-escalation studies in human and mouse cell models of Wolfram syndrome: monitor calpain 2 activation and cell death
2. Dose-escalation studies in animal models of Wolfram syndrome, both in brain-specific and beta cell-specific WFS1 knockout mice.
3. Pharmacokinetic studies
Thank you for your patience. I have been doing my best.
Kindly,
Fumi Urano