In the past two years, my team has identified drug targets, developed animal and cell models of Wolfram, and identified small molecules for treating patients with Wolfram syndrome. We completed key preclinical studies last week and submitted a research article to the National Academy of Sciences of the USA (Medical Science section) last night. I have been fortunate to have wonderful fellow scientists, physicians and technical assistants in my team. I felt so grateful. Based on these findings, we keep on moving forward to advance to our next step.
Here is the acknowledgment section of our article.
We thank all of the participants in the Washington University Wolfram Registry and Research Clinic and their families for their time and effort (https://wolframsyndrome.dom.wustl.edu/). We also thank Mai Kanekura, Mariko Hara, and Karen Sargent for technical support and the Washington University Wolfram Study Group Members and the study staff for advice and support in the greater research program. This work was supported by grants from NIH (DK067493, P60 DK020579, and UL1 TR000448), JDRF (47-2012-760 and 17-2013-512), ADA (1-12-CT-61), the Team Alejandro, the Team Ian, the Ellie White Foundation for Rare Genetic Disorders, and the Jack and J.T. Snow Scientific Research Foundation to F. Urano. We dedicate this article to late Dr. M. Alan Permutt, and Mr. Jonathon Wasson who devoted their lives to Wolfram syndrome research. Their deaths were a great loss for us all.
Thank you for reading this blog. I saw many videos of people participating in the bucket challenge for Wolfram syndrome. I appreciate your support. Thank you, Thank you, Thank you.
