Invitation to Individuals of Ashkenazi Jewish Descent for WFS1 Genetic Testing
Wolfram Syndrome Study: The goal of this study is to identify patients and carriers of the Jewish subtype of Wolfram syndrome and to characterize their natural history in order to guide the development of future genetic screening and clinical trial programs. You may be eligible to participate if you have Ashkenazi Jewish ancestry.
September 2025 Research Updates
Researchers at WashU Medicine are developing therapies for the Ashkenazi Jewish subtype of Wolfram syndrome, a rare inherited condition in which about one in every 30 – 40 people of Ashkenazi Jewish ancestry carries the WFS1 variant. The lab’s long-term goal is to find a cure, and current work focuses on gene-editing technologies that address the root cause of the disease rather than just managing symptoms.
The Washington University Lab Working to Cure Jewish Wolfram Syndrome
At Washington University School of Medicine in St. Louis, Missouri, a dedicated team of scientists is working to change the future for people living with Wolfram syndrome.
Led by Dr. Fumihiko (Fumi) Urano, MD, PhD, one of the world’s foremost experts on the condition, the lab has become a global center for research and clinical care, especially for families affected by the Ashkenazi Jewish variant of Wolfram syndrome.
Finding a Cure: New Research on the Ashkenazi Variant
Learn more about current research projects related to the Ashkenazi Jewish variant, and how to contribute to finding a cure. The following is a list of current research led by WashU Medicine. If you would like to make a donation to support this research, you may do so here.
Sign Up for the International Registry
If you or a family member has Wolfram syndrome and Ashkenazi Jewish heritage, your participation could help move research forward.
Washington University’s internationally recognized team is actively seeking new patients to join their clinical care and research efforts. Signing up for the Wolfram International Registry is the first step.
Published Research and Articles
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"A revealing flaw: A rare disease that cripples a key cellular organelle holds clues to treating more common conditions"
A 2021 Science article shows how breakthroughs in understanding Wolfram syndrome—caused by ER stress—are leading to new drug and gene therapies that may also aid in treating diseases like diabetes and Alzheimer’s.
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"A phase Ib/IIa clinical trial of dantrolene sodium in patients with Wolfram syndrome"
A 2021 study found that the drug dantrolene sodium is safe for people with Wolfram syndrome. While the drug didn’t improve vision or overall insulin production, it showed some potential for helping certain patients and reducing cell stress.
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"Multidimensional analysis and therapeutic development using patient iPSC–derived disease models of Wolfram syndrome"
Using stem cells, researchers showed that treatment with chemical chaperones improved cell health and increased insulin production, suggesting a potential therapy for the Ashkenazi subtype of Wolfram syndrome.