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 […]
Dr. Benjamin Delprat University of Montpellier, France In my lab, we are developing concomitantly two therapeutic strategies: a pharmacological approach and a gene therapy. To achieve these goals, we are working with suited animal models: two transgenic mouse lines and one zebrafish line. One mouse model and the zebrafish line are deficient for Wolframin, […]
Dr. Timothy Barrett’s Update | University of Birmingham, United Kingdom Dear friends and colleagues, I hope everyone is keeping OK. I have 3 items to update you on. Firstly, the TREATWOLFRAM trial is continuing to progress well. We had an independent Data Monitoring Committee meeting in January. This is chaired by Professor Karen Morrison, an […]
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 […]
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/
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!
Dr. Fumihiko Urano Washington University School of Medicine, USA Dear Friends, I want to take a moment to express my deep gratitude for your unwavering belief in and support of our mission to find a cure for Wolfram syndrome. Your enduring encouragement has been a beacon of hope guiding us on this remarkable journey. […]
Dr. Vania Broccoli San Raffaele Hospital/CNR-Institute of Neuroscience in Milan, Italy. Eye gene therapy for restoring WFS1 gene function in Wolfram mice AAV-based gene therapy is based on intra-vitreal injections of AAV vector expressing a therapeutic gene. This strategy is already in clinical use for treating Leber hereditary optic neuropathy (LHON) and clinical trials performed […]
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