Progress in the Past 15 Years of Wolfram Syndrome Research

October 14, 2024

Dear friends and colleagues,

In 2009, at the inaugural International Wolfram Syndrome Symposium in Paris, France, organized by the Association du Syndrome de Wolfram under the leadership of President Nolwen Le Floch (see attached picture taken in 2009!), we established ten key goals:

  1. Develop a patient registry
  2. Conduct a longitudinal study
  3. Identify biomarkers
  4. Create a multi-disciplinary service clinic
  5. Establish clinical guidelines
  6. Develop animal and cell models
  7. Identify drug targets and foster drug development
  8. Promote international collaboration
  9. Share data and reagents
  10. Find a cure

Thanks to the remarkable efforts of patient advocacy groups, families, physicians, and scientists worldwide, we have achieved nine out of these ten goals. The only remaining challenge is goal 10: finding a cure. However, we are making great strides—three clinical trials are currently ongoing, and gene-editing therapy and regenerative therapy are within reach.

We look forward to sharing some exciting announcements with you soon. Thank you for your continued support.

Sincerely,

Fumi

Fumihiko URANO, MD, PhD

Samuel E. Schechter Professor of Medicine

Washington University School of Medicine