Author: urano

Dear Friends, Thank you so much for your support. I hope you are enjoying this beautiful autumn season with your family, and it is time for me to update you about our progress. Today, I’d like to talk about one of the new drugs we are developing as we have made significant progress on this […]

I’d like to begin by thanking all of you for your continued support, trust, and faith in me. I always feel your support and energy. It is always my pleasure to update you about our progress. To achieve a cure for Wolfram syndrome, we need breakthrough treatments. We currently have three new candidate drugs that […]

US Senator Roy Blunt (Missouri) and Director of National Center for Advancing Translational Sciences/NIH Dr. Christopher Austin visited us from Washington DC on August 9th. I had a chance to present our Wolfram syndrome clinical and research program to their teams. My colleague in Internal Medicine, Dr. Dan Ory, and I tried to tell them […]

Another successful Wolfram research clinic was completed this week. I would like to thank all the participants, Dr. Hershey, Dr. Marshall, Mrs. Samantha Ranck, and other physicians and scientists who were involved in the clinic. See you next year! Thank you again. Warmest regards,Fumi Urano

I have received many questions regarding our ongoing clinical trials, as well as questions related to our next step lately. We started the patient registry, and then moved onto the research clinic. Now, we are conducting trials. I think we are making progress, and need to speed up as we are racing against time. Although we don’t […]

I have received many emails and phone calls regarding the ongoing clinical trials for Wolfram syndrome in the US, UK, and Europe. After the discussion with Dr. Barrett’s team in the UK and other regulatory experts, we decided to summarize the gist of the ongoing clinical trials. You can find the information on the following […]

Dear Friends, I would like to update you about the status of our clinical trial of dantrolene sodium in patients with Wolfram syndrome. Dantrolene is a US Food and Drug Administration (FDA)-approved drug currently used for different medical conditions. Based on our data, we received orphan drug designation of dantrolene sodium for the treatment of […]

Our current focus is to bring our potential drugs to our patients. We are interested in drugs that can help patients “regain homeostasis.” In other words, we try to help our patients’ organ systems to keep cellular endoplasmic reticulum states stable. The challenge is that there are not many drugs that can modulate endoplasmic reticulum […]

We have become one of the first  8 members of the NIH’s Rare Disease Data Repository Program. This is one of the important projects at the National Center for Advancing Translational Sciences (NCATS), National Institutes of Health (NIH) in the USA. I have also become a steering committee member of this program. The purpose of this […]

I tried to make progress in the following three areas in 2016: 1) Improve Clinical Care, 2) Raise Awareness, and 3) Develop Novel Treatments for Wolfram syndrome. I did my best. I know I could not make a breakthrough, but I will keep on trying. My highest priority right now is to work out the […]

I would like to accomplish the following three goals: #1 Improve Clinical Care, #2 Raise Awareness, and #3 Provide a Cure for Wolfram syndrome. I feel that we have been making steady progress in #1 and #2. #3 is the most challenging one. To achieve #3, we need to first delay the progression of the […]

Our annual Wolfram syndrome research clinic took place this week. I would like to thank all the participants, staff members, scientists and doctors who were involved in the clinic. Two clinical trials are planned in the US and UK. I will keep you updated about these. I always appreciate your continued support. Take care,Fumi Urano

I have been in Washington DC this week to visit the National Center for Advancing Translational Sciences (NCATS) of National Institutes of Health and attend the World Orphan Drug Congress. My trip has been quite fruitful so far. I had a productive discussion with the drug development team at NCATS. We do have new drug […]

I received many emails regarding the status of the drug development. I am sorry that I have not updated you through this site. I am making steady progress. Our current target is “endoplasmic reticulum (ER) calcium homeostasis.” Based on our preclinical studies, we need to maintain high ER calcium levels to delay the progression of […]

Today is a Rare Disease Day. I am thinking of our patients and families suffering from life-threatening diseases in the world. I am very sorry that I have been slow. I will keep on doing my best to develop and bring novel treatments to our patients. I always appreciate your continued supports. I understand that […]

I just came back from my trip to Denver. I attended the fundraising event hosted by the Ellie White Foundation in Boulder on the 17th. I was impressed and moved by Ellie and her mother’s speeches. I could connect with their friends, families, supporters, and other patients. I would like to thank the White family, […]

Last week, I gave a lecture on Wolfram syndrome at the University of Southern California Medical Center. I received constructive comments and feedback. I gave more than 100 lectures on Wolfram syndrome in the past 10 years. I will keep on doing my best to raise awareness of Wolfram syndrome. I was recently asked to […]

This year’s Wolfram Syndrome Annual Conference in UK was successful again. I could connect with patients and doctors in UK again. I learned a lot from them. I would like to thank Tracy Lynch, Paul Lynch, Alan Nye, Lottie Nye, Dr. Timothy Barrett, Dr. Patrick Yu Wai Man, Dr. Karen Morrison, and all the patients […]

My friend and colleague, Mrs. Tracy Lynch, posted the following comments on her FB site. Tracy is the Chief Executive of the Wolfram UK, the largest Wolfram syndrome patient organization in the UK.  Her daughter has Wolfram. I completely agree with you, Tracy. Kids with special needs are not weird or odd. They only want what everyone […]

I had an important meeting today. I did my best. I will find out the outcome soon. Thank you for your support, everyone. Warmly,Fumi Urano

This year’s Wolfram Syndrome Annual Conference in UK was successful again. I could connect with patients and doctors in UK again. I learned a lot from them. I would like to thank Tracy Lynch, Paul Lynch, Alan Nye, Lottie Nye, Dr. Timothy Barrett, Dr. Patrick Yu Wai Man, Dr. Karen Morrison, and all the patients […]

Are we making progress? Yes, we are. We have discovered FDA-approved drugs that can potentially delay the progression of Wolfram. We are actively conducting a trial using one of these drugs in one of our mouse models of Wolfram syndrome. The results look promising, but I have been trying to be cautious. Our collaboration with […]

Is headache related to Wolfram syndrome? This is a difficult question because most of us experience headache even though we don’t have Wolfram syndrome or other medical conditions. As far as I know, headache may be related to autonomic dysfunction or neuropathy in Wolfram. Some patients experience sharp and stabbing unilateral pain similar to trigeminal […]

Recurrent urinary tract infection (UTI) is one of the most common clinical challenges. The bladder dysfunction caused by the central and peripheral neurologic dysfunction is thought to be the underlying cause of UTI. Urine culture is recommended for Wolfram patients with fever or other symptoms, such as headache. Inflammatory conditions associated with UTI may cause […]

I gave a lecture on Wolfram syndrome at the Berrie Diabetes Center, Columbia University Medical Center, in NYC last week. I could connect with patients, doctors, nurses, clinical coordinators, and researchers in New York City. I was impressed by their enthusiasm for developing treatments for Wolfram syndrome. I could establish important collaborations there. Please stay […]

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 […]

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 […]

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 […]

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 […]