Blog

Dr. Barrett and I gave lectures on Wolfram syndrome at the Japan-Korea Diabetes Symposium in Japan this week. Our lectures were successful. I had a chance to speak with Japanese physicians who see patients with Wolfram syndrome. The president of Japanese Diabetes Association, Dr. Tanizawa, and the President of the American Diabetes Association, Dr. Dagogo-Jack, […]

I had a very productive meeting recently with a doctor from India. He came to see me because he has many patients with Wolfram syndrome in his clinic in India. I was so impressed by his dedication to his patients with Wolfram syndrome. We discussed many things including a joint clinical study. It is my […]

My current priority is to bring an existing drug that can control endoplasmic reticulum (ER) functions to our patients with Wolfram syndrome. However, this is not good enough to halt the progression and/or reverse symptoms. We need to design a new drug that is specifically designed for Wolfram syndrome. How can we achieve this? We […]

My highest priority right now is to repurpose existing drugs for treating our patients with Wolfram syndrome. In parallel, we need a marker that can be easily monitored every week. This marker will tell us if the treatment is working. We also need an assay to monitor the levels of each marker. My team has […]

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 […]

I receive many emails, letters, and phone calls every day from patients with Wolfram syndrome and unusual forms of diabetes and their families and doctors. Many of these are related to their medical conditions and potential treatments. Some of these are personal. I received a message from a mother who had lost her daughter due […]

April was a challenging month for us, but now we know how to overcome challenges and are getting better to find other solutions. Together with the Snow Foundation, we have figured out to continue our preclinical studies required for setting up an interventional study for Wolfram syndrome. I received so many emails last month when we […]

I have some good news to share with you. We have just joined the Global Rare Disease Registry Program of the National Institutes of Health (NIH)/National Center for Advancing Translational Sciences (NCATS). Dr. Austin, Director of NCATS, and Dr. McInnes, Director of the Office of Rare Disease Research, helped me to make this happen. Dr. Yaffa Rubinstein […]

We had a successful fundraising event yesterday in St. Louis hosted by Alex’s Army and the Snow Foundation. My family, the Gebel Family, and Dr. Naseer attended the event. We played bowling together and shared out thoughts. Thank you so much for coming to the event. Thank you, Thank you, Thank you. Take care, Fumi […]

My visit to NIH, NCATS, with Stephanie Snow Gebel, Chairman of the Board of the Snow Foundation, was quite successful. We have had multiple discussions in the past two weeks and agreed to develop novel chemical compounds (i.e., drugs) for Wolfram syndrome. We will target the endoplasmic reticulum. This will be a three-way collaboration with NCATS, […]

I feel that we are getting closer to the tipping point for developing treatments and establishing firm international collaborations for potential future clinical trials. I have been continuously working to make the medical research field tilt toward us, the Wolfram syndrome program. I keep on moving forward. I understand that you are facing challenges related to […]

We need to take action. Many doctors and scientists have great ideas to develop novel treatments for Wolfram syndrome and type 1 diabetes. Only a fraction of these ideas have been implemented and translated to save patients. We need to take action. We need to use the highest level of science to help patients. For […]