Tomorrow is already a day late

My major focus is to develop treatments and provide a cure for Wolfram syndrome. Because Wolfram is a prototype of ER stress-related disease, I am running projects related to other ER stress-related diseases, Type 1 diabetes and ALS, on the side because I can learn new things and come up with new ideas. I often […]

Thank you for your encouragement.

I have received many emails and FB messages in the past few days. It was certainly devastating that we could not get support from the Bear Cub Funds of the Washington University, but we should move on. One of the major reasons we did not get support this time was that Wolfram is an ultra […]

Partnership with our patients

What is important to develop new treatments and achieve a cure for Wolfram syndrome? “Partnership with our patients”  We should know what our patients need and want, and our patients and their families and supporters should know and understand our strategies and plans. To achieve a cure, we need to work as a team and create […]

We did not get the Bear Cub Fund, but please don’t worry

I received many emails regarding the status of my application for the Bear Cub Funds from Washington University. Although I was one of the finalists, I did not get the fund. I apologize for not getting this fund to complete the preclinical study and set up a clinical trial for Wolfram syndrome. When I heard […]

Renovation of an old drug

One of the exciting things that were offered during our visit to NIH, NCATS, was to renovate old drugs and make them more specific for Wolfram syndrome and other endoplasmic reticulum-related diseases. We have identified three FDA-approved drugs that can potentially delay the progression of Wolfram syndrome. These drugs were originally designed and indicated for […]

Our visit to NIH was fruitful!

Stephanie Snow Gebel, the founder of the Snow Foundation, and I visited NIH, National Center for Advancing Translational Sciences, yesterday. Our visit to NIH was quite fruitful. I learned a lot about the NIH’s efforts on developing novel treatments for rare diseases. I met with Dr. Christopher Austin’s team and discussed our potential collaboration on Wolfram […]

Ready for my presentations at NIH

I will give two presentations at NIH tomorrow, 1) Endoplasmic Reticulum Disease Center and 2) Wolfram Syndrome Clinical Trials and Drug development. I am ready for these two presentations. I will do my best to get support from NIH to develop novel treatments for Wolfram syndrome and related disorders, and bring these treatments to our […]

Precision medicine and rare disease

Dr. Francis Collins is a renowned physician scientist, Director of National Institutes of Health, and my role model. I was watching his interview by Dr. Eric Topol last night, and came up with multiple ideas. Visit Site http://www.medscape.com/viewarticle/841877?src=sttwi Based on the technologies available for us including genome sequencing, we can potentially figure out molecular wiring […]

Bear Cub Fund from Washington University

I will find out the outcome of the Bear Cub Fund early next week. Please wish me luck. The money will be used to complete our preclinical studies to set up the clinical trial for Wolfram syndrome. We really need to get the full amount. I am praying. Looking forward with fingers crossed, Fumi Urano

Our visit to San Francisco was successful

Our visit to San Francisco was successful. We had multiple meetings and tried to get funds for research on Wolfram syndrome. Dr. Saad Nasser, Stephanie Snow Gebel, Anne Harkin, JT Snow, and myself did our best. I have no regret. I feel that something big is coming to us. I appreciate your continued support. Take […]

San Francisco Day 1

I came back to San Francisco with the Snow Foundation. Our first day was productive. We will meet with different types of people to accomplish our goals, Raise Awareness, Improve Clinical Care, and Develop Novel Treatments for Wolfram syndrome. I feel that something wonderful is going to happen to us here. I am hopeful. I […]

Genome editing: We should participate in the discussion

I was reading the NY Times today and found out that some scientists suggested that we should be careful about using the new genome editing technology. The article says, “The biologists fear that the new technique is so effective and easy to use that some physicians may push ahead before its safety can be assessed. […]