2018 goals
I thought a lot about our therapeutic development for Wolfram syndrome in the past several weeks. I feel that drug-repurposing may not be good enough. I firmly believe that we need a breakthrough therapy for Wolfram syndrome. This year, I would like to spend more time for developing regenerative gene therapy, especially for visual impairment. […]
Ready to say goodbye
Thank you for reading my blog. I always feel your support. I appreciate it immensely. As I am ready to say goodbye to 2017, I would like to update you about our progress. Our drug-repurposing clinical trial of dantrolene sodium is ongoing. The trial began in January, and nineteen patients with Wolfram syndrome from the […]
US Senator Blunt and NCATS Director Dr. Austin visited us from DC
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 […]
Thank you, Wolfram Research Clinic
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
Clinical trial update: next phase
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 […]
Clinical Trials in the US, UK, and Europe
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 […]
Clinical trial update
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 […]
Homeostasis
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 […]
Become a member of the NIH’s Rare Disease Program
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 […]
Thank you for your continued support.
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 […]
Wolfram UK Conference and their Clinical Trial plans
I attended the Wolfram UK and WellChild conference in England on Saturday, October 8th, 2016. This was my third time at the conference and met with around 100 patients and their family members. At the conference, Dr. Timothy Barrett (University of Birmingham) announced that his phase 2 randomized clinical trial of his repurposed drug in patients […]
Goals and progress in August 2016
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 […]