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.
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