Dr. Urano's Blog

Clinical Trials

A Proof of Concept

We are trying to show the proof of concept for developing novel therapies for Wolfram syndrome. What is a “Proof of Concept”? A proof of concept is a demonstration or verification that our theory has the potential for the real-world application. It our case, we have to show that the modulation of endoplasmic reticulum calcium homeostasis is beneficial for Wolfram syndrome patients. This requires a clinical trial. Once we have evidence that the modulation of ER calcium homeostasis is beneficial for real patients with Wolfram syndrome, this will open up many possibilities for patients.

Thank you for reading this blog. I hope you have a wonderful weekend.


Fumi Urano