June 28, 2018

Dear Friends and Supporters,

It is nice to see you again. I always appreciate your support, trust, and faith in me. I see patients with Wolfram syndrome every week and receive phone calls and emails from patients and their families every single day. It is definitely urgent to slow the progression of the disease and develop novel treatments for achieving a cure.

I attended two important meetings in the past two weeks. From June 11th to June 12th, I attended the International Wolfram Syndrome Workshop in Paris, France, hosted by the French Wolfram Syndrome Association. I attended the first workshop in 2009 together with just 11 other researchers from all over the world. I was the only researcher from the US then. At the first workshop, we agreed to create patient registries and research & service clinics for future clinical trials. As of today, these goals have been achieved, and two drug-repurposing clinical trials are ongoing (https://wolframsyndrome.dom.wustl.edu/clinical-trials/). I met with late Dr. Alan Permutt from Washington University and Mrs. Stephanie Snow Gebel who founded the Snow Foundation at the workshop in 2010, which made me decide to move to Washington University in 2012. At this year’s workshop, almost 50 researchers attended and presented their progress. I presented our progress on our clinical trial and gene therapy strategies. My colleague, Dr. Tammy Hershey, presented MRI findings in patients with Wolfram syndrome. My long-term collaborator, Dr. Tim Barrett in the UK, presented his progress on their clinical trial. I met with multiple collaborators and saw representatives of patient organizations from US, UK, Italy, Spain, Belgium, and France. I was glad to see that the International Wolfram research group had grown. I was glad that all the researchers had been collaborative. I was grateful for everything patient organizations had done for our patients and families, doctors, and researchers.

On Monday, June 25th, I presented my concept of Wolfram spectrum disorder at the American Diabetes Associationmeeting in Orlando, Florida. I believe there are at least three different types of clinical manifestations in Wolfram syndrome and Wolfram-like disorder: mild, intermediate, and severe. I have been developing genetic testing to further understand the spectrum of Wolfram syndrome. Some of our new treatments could be beneficial for patients with all three types in theory. We also need to develop specific treatments for each type.

As always, please feel free to contact me with any questions or concerns (urano@wustl.edu). I would like to know what you think and how you feel. Thank you again for your support. Good things happen to people who do good things.

With passion, unity and gratitude,

Fumi Urano