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Posted On: 10/14/2024 11:52:38 PM
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Dr. Urano October Update
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:
Develop a patient registry
Conduct a longitudinal study
Identify biomarkers
Create a multi-disciplinary service clinic
Establish clinical guidelines
Develop animal and cell models
Identify drug targets and foster drug development
Promote international collaboration
Share data and reagents
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
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:
Develop a patient registry
Conduct a longitudinal study
Identify biomarkers
Create a multi-disciplinary service clinic
Establish clinical guidelines
Develop animal and cell models
Identify drug targets and foster drug development
Promote international collaboration
Share data and reagents
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
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