I always feel that we should raise awareness of rare diseases to provide a cure. We certainly need new therapeutics. We need small molecules to halt the progress. We need stem cell-based and genome-engineering-based therapies to provide a cure. My team has been working on small molecules, stem cells, and genome editing. However, we need to raise awareness of the disease. To bring our new treatments to our patients, we need funds and supports from the FDA and our local IRBs. To get support, we need to raise awareness. The Jack and JT Snow Foundation, the Ellie White Foundation, the Team Alejandro, the Team Ian, the Wolfram syndrome patient support group, the Wolfram syndrome UK, and association du syndrome de Wolfram are working hard to raise awareness. Please help us spread the word. http://wolframsyndrome.dom.wustl.edu/
Thank you for your encouragement and continued support. I hope you have a wonderful weekend. Kindly, Fumi Urano
