Are we making progress? Yes, we are.

  1. We have discovered FDA-approved drugs that can potentially delay the progression of Wolfram. We are actively conducting a trial using one of these drugs in one of our mouse models of Wolfram syndrome. The results look promising, but I have been trying to be cautious.
  2. Our collaboration with the drug development team at National Institutes of Health/NCATS has been going very well. We have identified new drugs for Wolfram syndrome. Further preclinical studies are needed and I am quite hopeful.
  3. The development of regenerative therapy using a biologic called MANF is ongoing. We are getting positive preclinical data.

To further develop these projects and bring new therapies to our patients, I need more funds. I have been writing multiple grant applications. The Snow Foundation has been working tirelessly to raise funds for my research. I also appreciate donations from the Ellie White Foundation, as well as other patient organizations, families, friends, and others. I want to save our patients. Please help me, Stephanie Snow Gebel, and Dr. Saad Naseer to raise funds.

Thank you for reading my blogs. I always appreciate your support.

Take care,

Fumi Urano