Who can help us bring novel treatments to our patients?

I ask this question to myself every single day. The answer is “ourselves.” We should create the strong community and get out together to ask for help (i.e, money). We have drug targets. We have a potential drug approved by FDA. If we have two million dollars right now, we can complete a preclinical study and start an interventional study. The clinic and genetic testing can help patients, but cannot save them. Novel treatment will save them. We need to develop novel drugs for Wolfram syndrome. This happened to patients with AIDS. It should happen to patients with Wolfram.

Take care,

Fumi Urano