Collaboration has always been at the heart of my approach to Wolfram syndrome research. It is collaboration, not competition, that drives us closer to our mission of achieving a cure for this devastating disease. Since 2010, I have actively participated in and helped organize the International Wolfram Syndrome Symposium. Our 9th symposium took place in Windsor, UK, from Tuesday, October 22, to Thursday, October 24. Remarkably, 14 years have passed since the first symposium in France, and only three of us have attended every single one: Dr. Timothy Barrett from England, Dr. Gema Esteban from Spain, and myself.
Over the years, we have seen many scientists and doctors join us at different stages of this journey. Some were involved from the beginning, others joined in the middle, and I was especially pleased to welcome new scientists and doctors at this year’s meeting. Our discussions spanned a wide range of topics, from cutting-edge advancements in gene therapy, regenerative therapy, and the development of oral medications and supplements, to foundational scientific research aimed at understanding the disease mechanisms. One significant outcome of our discussions is a plan to create clinical guidelines for the diagnosis and management of Wolfram syndrome.
Our collective understanding of Wolfram syndrome has deepened considerably over the past 14 years. Today, multiple clinical trials are underway—a testament to how far we’ve come. Yet, there is still much work to be done to achieve our ultimate goal. Progress requires continued effort, persistence, and, most importantly, collaboration.
I am excited to share that I have established new collaborations with research groups in Estonia, England, and Belgium. I will provide more details about these partnerships in my upcoming blog posts. From the United States, we were fortunate to have the participation of Stephanie Snow Gebel and Dr. Sarah Gladstone from the Snow Foundation, as well as Dr. Lahar Mehta, Dr. Nathalie Erpelding, and Dr. Ryan Miller from Amylyx Pharmaceuticals. I am deeply grateful to everyone who attended the symposium and contributed to the exchange of knowledge and ideas. I would like to thank the organizers of the meeting, the Snow Foundation, and Wolfram UK for their support and coordination.
I look forward to our next gathering in Paris, France, in 2025. The progress we’ve made so far gives me hope that, together, we can continue to push the boundaries of what is possible for those living with Wolfram syndrome.
Fumihiko (Fumi) Urano, MD, PhD
Director, Wolfram Syndrome and Related Disorders Clinic and Research
Washington University Medical Center