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 with Wolfram syndrome would start next year at multiple sites in the UK and Europe. I saw many patients were crying and was quite moved by the news. It was also announced that Mrs. Tracy Lynch and Mr. Paul Lynch from the Wolfram UK had received the UK Prime Minister’s Points of Light award for their contribution to raising awareness of Wolfram syndrome. All the participants applauded their wonderful accomplishments.
I presented that my phase 1b clinical trial of dantrolene sodium (drug repurposing: the drug is currently approved for the treatment of other medical conditions) in patients with Wolfram syndrome would start soon in the US with the support of the Snow Foundation and Ellie White Foundation. I felt that I was a little behind because our trial would be a smaller and shorter one. Dr. Barrett and Wolfram UK team offered help to conduct large scale multi-center trials together with us in the near future. So I felt grateful.
I was encouraged and motivated after the conference. Thank you so much for inviting me again. We will keep on working together.