Research News

Dr. Urano’s November Update

November 4, 2017 Dear Friends, It has been a while since we last talked. I hope you are enjoying this Fall season with your family and friends. I think about the value of family and friends a lot lately. Support from family and friends keeps me going. So I appreciate your continued support, encouragement, and […]

A British Consortium wins the joint call for proposals

Three British and one Australian research teams, coordinated by Professor Timothy Barrett, have been awarded the funding of 200,000 € by the Association syndrome de Wolfram, the Eye Hope Foundation and the Snow Foundation. Their project aims at “developing biomarkers that will show early evidence of benefit of a treatment; and patient self-report outcome measures […]

Dr. Urano’s September update

September 22, 2017 Dear Friends, It is nice to “meet” you again. Thank you for your generous and continued support for our therapeutic development for Wolfram syndrome. With the support from the Snow Foundation, multiple patient organizations and supporters around the world, and federal grants, I could maintain the Wolfram syndrome program to study the […]

Dr. Urano’s August update

Dear Friends, I hope you had a wonderful summer season. I would like to update you about our progress. Our clinical trial of dantrolene sodium in patients with Wolfram syndrome is ongoing. We have been monitoring the safety, tolerability, and efficacy of the drug. Some patients have been taking dantrolene sodium for more than 6 […]

Washington University Wolfram Research Clinic completes its 8th consecutive year!

Dear Wolfram Families and Supporters: The Washington University Wolfram Research Clinic (or ‘natural history) study just finished its 8th consecutive year of assessing patients with Wolfram Syndrome. This work began in 2010, supported in part by the Snow Foundation. For the past 5 years, it has been supported by a National Institutes of Health grant […]

TreatWolfram Clinical Trial update – June

Dear Friends and colleagues, I would like to give you an update as to progress with our clinical trial of a treatment for Wolfram syndrome. We had the final confirmation of funding from the UK Medical Research Council on December 16th 2016, so were able to start work on the study protocol early in the […]

Dr. Urano’s June Wolfram Research Update

Dear Friends, It’s a spectacular Saturday morning in Saint Louis as I write this to you. I always appreciate your continued support, encouragement, and kind words. I have received many questions regarding our ongoing clinical trial, as well as questions related to our next step lately. Although we don’t have a concrete plan yet, we […]

Dr. Barrett meets with Wolfram Associations across the globe

Dear Friends and colleagues, I have been fortunate to visit the French Wolfram Association meeting in April in Paris; and the Spanish Wolfram association meeting in May in Almeria. I was humbled by the friendship and community spirit of these families; and they were very tolerant of my poor French and Spanish language skills. On […]

Research update letter about treatment studies in Wolfram Syndrome


Clinical Trial Update by Fumihiko Urano, M.D.

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 […]