Entries by The Snow Foundation
Our drug-repurposing Clinical trial of Dantrolene Sodium-Dr. Fumihiko Urano, MD, PhD
Our drug-repurposing clinical trial of dantrolene sodium in patients with Wolfram syndrome has been almost concluded. Nineteen patients could successfully complete the required six-month phase, and many of them decided to stay on dantrolene sodium another 18 months. The results of this open-label clinical trial (all the participants took dantrolene sodium) show that dantrolene sodium […]
New Genetics Clinic for Wolfram syndrome/Wolfram-related disorders-Dr. Fumihiko Urano, MD, Phd
To further improve the clinical care for patients with Wolfram syndrome and Wolfram-related disorders, I have created a new genetics clinic at Center for Advanced Medicine, Washington University Medical Center. We offer genetic evaluations, education, and counseling for patients and family members of all ages with or suspected to have Wolfram syndrome or WFS1-related disorders. […]
Base Editing Gene Therapy- Dr. Fumihiko Urano, MD, PhD
In collaboration with Dr. David Liu’s team at Harvard University/Broad Institute and Dr. Catherine Verfaillie’s team at the Katholieke Universiteit Leuven, we have been developing a novel gene therapy called Base Editing for Wolfram syndrome. This technology uses some components from CRISPR systems together with other enzymes to directly replace abnormal WFS1 gene with normal […]
Regenerative Gene Therapy- Dr. Fumihiko Urano, MD, PhD
My current focus is to develop gene therapy for Wolfram syndrome. Our ultimate goal is to provide a cure using regenerative gene therapy. We have been trying to improve diabetes, visual acuity, and brain functions using viral vectors of a healthy Wolfram gene and a regenerative factor called MANF in mouse models. We are getting […]
Novel Drugs for Wolfram Syndrome-Dr. Fumihiko Urano, MD, PhD
We are aware that a drug-repurposing is not the best approach to halt the progression of Wolfram syndrome. We need cutting-edge treatments designed explicitly for Wolfram syndrome. Based on the clinical trial data of dantrolene sodium in patients with Wolfram syndrome, we have been actively developing novel drugs in collaboration with the drug development team […]
Dr. Neil White & Dr. Bess Marshall
Washington University School Of Medicine The study of liraglutide (Victoza) in Wolfram Syndrome that is being conducted by Drs. White and Marshall at Washington University has gotten off to a slow start because of the COVID-19 pandemic. However, they have now recruited 10 subjects who are in various stages of recruitment, screening or treatment. Two […]