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A cure for Wolfram syndrome could lead to a cure for diabetes and blindness.

Sunday, August 10, 2014

Dose escalation study?

Recently, I often speak with medical and scientific officers of pharmaceutical industries. On Thursday, I had an exciting discussion with several officers of a pharmaceutical company. On Friday, I had another fruitful discussion with an expert in R&D, and Regulatory & Clinical Trials. I have a much better idea of what I should do to perform interventional studies for Wolfram syndrome and Type 1 Diabetes. 

What is most important? The answer is a "small molecule." We need a small molecule (i.e., a drug) to perform a clinical trial. This is probably obvious to anyone. We need another important component, the dose. We need to establish the recommended dose. There are multiple ways to accomplish this, and I am learning these.

I hope you will have the best Sunday in your life!