Regenerative medicine 101
Our Regenerative Medicine 101 event on Thursday was successful. We could raise awareness of regenerative medicine. I would like to thank Dena Ladd at Missouri Cures and Lila Solnica-Krezel and Angela Bowman at Center of Regenerative Medicine at Washington University Medical Center for organizing this important event. Here is my presentation. Please feel free to […]
Making steady progress
Three things are always on my mind: 1) Improve Clinical Care, 2) Raise Awareness, and 3) Provide a Cure for Wolfram syndrome. Our current focus is to “repurpose” one of FDA-approved drugs for the treatment of patients with Wolfram syndrome. We have been making steady progress on this, and things are looking good at the […]
Interventional studies
As I mentioned repeatedly in my previous blogs, my highest priority right now is to start interventional studies using currently approved drugs. This strategy is called “drug repurposing.” This is an attractive therapeutic option because the development of novel therapeutic compounds takes a lot of time and money, which can be avoided by the use of readily […]
Wolfram Night at Busch Stadium Thank you, Cardinals and SF Giants!
We had a wonderful night at Busch Stadium raising awareness for Wolfram syndrome together with the on Monday, August 17th. Raquel Gebel, our patient with Wolfram syndrome, and I got to throw out the 1st Pitch before the Cardinals took the field. Raquel did a wonderful job. I would like to thank all the people who […]
Two lectures on juvenile diabetes in NYC in September
I have three goals in my mind all the times: 1) Improve Clinical Care, 2) Raise Awareness, 3) Develop Novel Treatments and Provide a Cure for Wolfram syndrome. I believe that our novel treatments for Wolfram syndrome can lead to a cure for other forms of diabetes, including Type 1 diabetes. I have been invited […]
Connecting patients: NIH Global Rare Disease Registry Program
As I mentioned before, our Wolfram syndrome International Registry and Clinical Study has been selected to participate in the NIH Global Rare Disease Registry Program. Fortunately, I have been appointed to serve as a steering committee member for this important initiative. We would like to accomplish the following goals through this program. For patients and […]
Regenerative medicine to combat juvenile diabetes and blindness
My current focus is to delay the progression of Wolfram syndrome using small compounds (i.e., pills), especially repurposing currently available FDA-approved drugs. I am aware that this is not good enough because many of our patients have lost tissues producing insulin, maintaining brain functions, and preserving visual acuity. In collaboration with biomedical engineers and biotechs, […]
Genetic testing and genome sequencing
Here is a question that I often get. “Should we get our child’s genome (i.e., the entire DNA information) sequenced?” This is a challenging question because my answer to this question may change based on the development of our technology. As of today, my answer is “No.” The contrast between our ability to identify genetic […]
Wolfram research clinic–Thank you
Today’s was the last day for the Wolfram research clinic 2015. I would like to thank all the participants, their families, physicians, researchers, and staff members who have been involved in the clinic. I cannot thank you enough. Our next step is to start an interventional study. I will keep on working. We are racing […]
Fundraising event at Manna by the Ellie White Foundation
We had another fundraising event at Manna in Wilmington on July 8th. This was hosted by the Ellie White Foundation. I was gratified to know that the event was successful. My research for the therapeutic development for Wolfram syndrome has been supported by multiple patient organizations. I would like to thank everyone who attended the […]
Thank you, JT Snow
Our trip to San Francisco this week was quite successful. I flew into San Francisco with Mrs. Stephanie Snow Gebel and Dr. Saad Naseer on Tuesday and had multiple meetings with biotech executives and others. Our fundraising events were quite successful. JT Snow, a renowned former SF Giants player and Stephanie’s brother, helped us a […]
International network of doctors
International Network of doctorsI have been trying to expand the network of doctors who see patients with Wolfram syndrome. It is becoming international. Dr. Iwasaki in Tokyo, Japan, has agreed to be a point-of-contact doctor in Tokyo. She has a few patients in her area. Please feel free to send me email in Japanese (urano@dom.wustl.edu) […]