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Dr. Fumihiko Urano | Washington University School of Medicine, USA | Prilenia Therapeutics


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.

Read more

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AMYLYX PHARMACEUTICALS Helios Update

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.

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Thank you to McCarthy Building Company for raising money for TSF

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!!!!

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2024 Support Drive, could you help?

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/

Paypal-donate-QR-Code

 

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SAVE THE DATE APRIL 13TH, Wolfram Syndrome Global Virtual Conference RESEARCH UPDATES

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.

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Quest for a Cure across the globe

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!

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

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

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Donate today for Rare Disease Day

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

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Rare Disease Day is February 29th

Rare Disease Day is February 29th. Please help the Snow Foundation raise some awareness and funding.  Our goal is to raise $5,000 this month.  Please share and start your fundraiser on Facebook today, donate online

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Wolframin and SERCA – Molecular Partners

Publication: ncbi.nlm.nih.gov | Publication Date: September 30, 2014

Authors: Zatyka, M., Da Silva Xavier, G., Bellomo, E. A., Leadbeater, W., Astuti, D., Smith, J., Michelangeli, F., Rutter, G. A., & Barrett, T. G.

Abstract

Wolfram syndrome is an autosomal recessive disorder characterized by neurodegeneration and diabetes mellitus. The gene responsible for the syndrome (WFS1) encodes an endoplasmic reticulum (ER)-resident transmembrane protein that is involved in the regulation of the unfolded protein response (UPR), intracellular ion homeostasis, cyclic adenosine monophosphate production and regulation of insulin biosynthesis and secretion. Read more

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