Dr. Urano's Blog

8 drugs-Mechanism of Action?

Here is a brief summary of our small-scale drug screen to identify novel drugs for Wolfram syndrome.

  • We have identified 8 drugs.
  • 4 out of 8 are FDA approved drugs for other indications.
  • 3 out of 4 seem to have the same mechanism of action

What is the “mechanism of action”? It refers to the targets on which a drug interacts (receptors and enzymes) as well as the particular way of action (inhibitors or activators). In our case, these 3 drugs target the same enzyme (i.e., drug target) and inhibit the action of the enzyme.


“Our article on the discovery of this novel drug target and potential drugs for Wolfram syndrome has been accepted for publication. We will show our preclinical data.” The press embargo on our article¬†will lift on November 24, 2014 at 3:00 PM U.S. Eastern time.