Regenerative medicine symposium
I had a chance to present my data and concept on regenerative medicine for the treatment of Wolfram syndrome last week at the regenerative medicine symposium in Tokyo, Japan. Five faculty members from Washington University attended this important symposium. I got constructive feedback from the audience. To achieve a cure, we need to develop regenerative […]
Thank you for the Wolfram Research Clinic
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
Wolfram Syndrome Workshop in France
I attended the 6th Wolfram syndrome workshop in Nantes, France last week. I still remember the first workshop which was held in 2009. Since then, we have made significant progress in the past 7 years. Two potential clinical trial plans in the US and UK were discussed. I presented my ideas and plans about a […]
Washington DC, NCATS, and World Orphan Drug Congress
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 […]
Keep on working
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 […]
Rare Disease Day
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 […]
A fruitful trip to Denver
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, […]
Thank you, Wolfram syndrome patient organizations in the world
The rare disease day is approaching. I would like express my deep appreciation and gratitude to multiple patient organizations supporting my research. I will keep on working with them to improve clinical care, raise awareness, and develop novel treatment for Wolfram syndrome. Snow Foundation (USA) Ellie White Foundation for Rare Genetic Disorders (USA) Worldwide Society […]
100 lectures on Wolfram in the past 10 years
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 […]
Climb the stairs together hand in hand
I would like to begin by thanking all the people around the world who keep on supporting me. I think of you all the time. We climb the stairs together hand in hand to bring novel treatments to our patients. Thank you again. I will not give up. I will keep on doing my best. […]
Thank you, Thank you, Thank you. Our progress.
As we approach the end of this year, I would like to thank you again for your continued support, encouragement, and patience. The number of emails and phone calls I receive has been increasing. Three things are always on my mind.1. Raise Awareness of Wolfram Syndrome2. Improve the Clinical Care3. Provide Novel Treatments and A […]
Snow Foundation’s Wolfram Syndrome Clinical Trials Fund
The Snow Foundation has created the Wolfram Syndrome Clinical Trials Fund. I just looked at videos of our patients and was quite moved. I appreciate their continued support. I cannot thank you enough. We are racing against time.https://snowfunds.org/web/snow/pub I hope you have a wonderful weekend. Take care, Fumi Urano