2014 and HOPE for 2015

In 2014, I thought about three things every single day.

1. Improve clinical care for patients with Wolfram syndrome
2. Develop treatment options for delaying the progression of Wolfram syndrome
3. Raise awareness of Wolfram syndrome

In addition, I thought about regenerative therapy options for Wolfram syndrome and Type 1 diabetes, and my team conducted preclinical studies on this. I hope we will accomplish #2 and focus on regenerative therapies.

I feel that we have made progress in each area, but there is still a lot of room for improvement.

1. Could not figure out the underlying causes of some of the symptoms.
In short, I could not answer some of the questions from our patients and physicians. We need to know more about the disease.

2. Could not start a clinical trial.
We were close, but need more preclinical studies. I understand we are racing against time. Some patients passed away this year, which was devastating to me.

3. Could raise awareness
I have been closely working with the Snow Foundation and the patient support group in the US. In 2015, I hope I can extend this collaboration and work with patient support groups in Europe, South America, and Asia.

Thank you for reading this blog. I believe we have HOPE. I hope you have a wonderful 12/31.

Take care,

Fumi Urano