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 […]
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. […]
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 […]
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 […]
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 […]
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 […]
Catholic University of Leuven, Belgium Genome editing for Wolfram syndrome: Although rare individually, genetic disorders collectively constitute a common health problem. As the cause of these diseases is a defective gene, gene therapy would be able to resolve all of these disorders. Wolfram syndrome is a genetic disorder, with the ultimate symptoms of Diabetes, blindness […]
Update January 26, 2024: Dear Friends, Thank you sincerely for your invaluable support of our collaborative study alongside Prilenia Therapeutics. As you are aware, our mission to find a cure for Wolfram syndrome involves three crucial steps: Step 1: Slowing down the progression of the disease through the use of oral medications. Step 2: Halting […]
Update March 5, 2024 Our focus is to discover preclinical Liraglutide and the rest of the GLP1 receptor agonists available on the market to help stop the progression of Wolfram syndrome. Moreover, we are looking into a new generation of GLP1 and GIP co-agonists for the same reason. We are also developing gene therapy against […]
University of Cambridge, United Kingdom We are currently focusing our efforts on the following research areas. Disease progression and biomarkers We are collecting a comprehensive range of ophthalmological data on individuals who have been confirmed to carry pathogenic WFS1 mutations, including high-resolution optical tomography tomography imaging and visual electrophysiology. We want to carefully document the […]
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