Dr. Urano's Blog

Three step formula to provide a cure for Wolfram syndrome July 20, 2014

I presented my strategy for providing a cure for Wolfram syndrome yesterday. I think there are three steps to achieve this.

1. Stop the progression
2. Protect and Regrow remaining tissues
3. Replace damaged tissues

Our current focus is to “Stop the progression” of the disease. We are testing if FDA-approved drugs currently used for other diseases may be beneficial for Wolfram syndrome patients, and we have four candidates. We are also developing new drugs specifically designed for Wolfram syndrome.

We plan to use MANF for protecting remaining tissues, especially eye cells. Our final step is to replace damaged tissues using regenerative medicine. I will keep on talking about these.