Dr. Fumihiko Urano

Washington University School of Medicine, USA

Dear Friends,

 

Thank you sincerely for your invaluable support of our collaborative study alongside Prilenia Therapeutics. 

One promising candidate is the Sigma 1 receptor, and we have initiated a collaboration with Prilenia, a biotech company specializing in this field. Together, we are investigating the effects of a drug called pridopidine, which targets the Sigma 1 receptor, in cellular models of Wolfram syndrome. We are grateful to the Snow Foundation in collaboration with Ellie White Foundation for their generous donations, which enables us to conduct this crucial study using cells derived from Wolfram syndrome patients. The study is currently in progress as we explore various experimental conditions to assess the efficacy of pridopidine. We are committed to providing you with regular updates on our progress.

 

Once again, we extend our heartfelt thanks for your generous support, which is instrumental in advancing our mission to find a cure for Wolfram syndrome and bring hope to those affected by this condition.

 

With grace and gratitude,

Fumi

 

Fumihiko Urano, MD, PhD, FACMG

Professor of Medicine and of Pathology & Immunology

Samuel E. Schechter Endowed Professor in Medicine

Director, Wolfram Syndrome/WFS1-related disorders Registry & Clinical Study and WFS1 clinic at BJC HealthCare

Washington University School of Medicine

https://wolframsyndrome.wustl.edu/

AMYLYX PHARMACEUTICALS
Helios Update

Earlier in February 2024, Amylyx Pharmaceuticals announced that enrollment was completed in the Phase 2 HELIOS clinical trial of AMX0035 (sodium phenylbutyrate and taurursodiol) for the treatment of Wolfram syndrome.

HELIOS is a 48-week exploratory, open-label proof of biology study assessing the effect of AMX0035 on a number of measures, including pancreatic function (mainly pertaining to diabetes), visual function, and Wolfram syndrome disease rating instruments.

The study, being conducted at Washington University in St. Louis, has enrolled 12 adult participants living with Wolfram syndrome, and preliminary results are anticipated in the second half of 2024. Data from this initial study will play an important role in giving Amylyx information needed to make decisions for potential future trials. Amylyx looks forward to sharing the results with the community, including at scientific meetings, when they are available.

McCarthy Building Company’s annual Dan Licari Memorial Fishing Tournament raised $13,445.00 for the Snow Foundation to support the Bieser family in their fight against Wolfram Syndrome.. THANK YOU!!!!

2024 Support Drive, could you help?

This year, we are funding four projects worth almost $400,000.
Please donate today to support our community. Your gift will make a significant difference and help us achieve our goal.
For More Information: https://thesnowfoundation.org/2024-support-drive-could-you-help/

 

https://thesnowfoundation.org/donate/

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Join the virtual conference The Snow Foundation and Wolfram Syndrome UK will host on April 13th.The conference will feature four presentations starting at 2:00 pm (BST). Details about the program and registration links will be forthcoming. This bi-annual event aims to educate everyone about the latest research on Wolfram Syndrome.

The Snow Foundation is grateful for the support it has received from people around the world. We would like to express our appreciation to Markus Klasenar for putting us on the map in Germany. Thanks to their fundraising efforts, we have raised 2,650 euros for our Quest for A Cure!

Mark your calendar! Dr. Fumihiko Urano to Present on Rare Disease Day at NIH 2024

Dr. Urano received an invitation to present his research on Wolfram Syndrome at the Rare Disease Day event held at the National Institutes of Health on February 29, at 2:20 Eastern Time. This event is widely regarded as one of the most prestigious gatherings for rare diseases, offering an excellent platform for us to raise awareness about Wolfram Syndrome. You can participate by watching his presentation remotely. The link to access it: https://ncats.nih.gov/news-events/events/rdd

The true power of giving is the ability of our supporters to leverage their own personal networks. Help us raise $5,000 this month for Rare Disease Day, February 29th. Donate online