The secret formula for developing new therapy

Our next step to find a cure for Wolfram syndrome was to set up a Wolfram clinic. A clinic was established to meet annually to obtain baseline measurements of pancreatic beta cells and neural dysfunction on a sample of patients in preparation for clinical trials of new therapeutic agents (i.e. drugs). We also started producing induced pluripotent stem cells using skin from patients. Pluripotent stem cells from patients will be used to test new drugs.

Here is the three step formula for finding a new therapy.

1. Understanding the disease

2. Identify and verify new therapeutic agents

3. Treatment of patients

I call it UIT (You and I Together)!