Entries by The Snow Foundation

Gonadal function in males with WFS1 spectrum disorder (Wolfram syndrome)—A European cohort perspective

Publication: onlinelibrary.wiley.com | Publication Date: 29 April 2025 Authors: J. Rohayem and O. Cunningham Abstract Background WFS1 spectrum disorder, also known as Wolfram syndrome (WS) is an ultra-rare (<1:500,000; ORPHA: 3463) monogenic (OMIM #222300) progressive neuroendocrine and neurodegenerative disorder, characterised by early-onset insulin-dependent diabetes, optic atrophy, central diabetes insipidus and sensi-neuronal deafness. It is caused […]

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Positive Long-Term Results from Phase 2 HELIOS Clinical Trial of AMX0035 in People with Wolfram Syndrome

Amylyx Pharmaceuticals, Inc. today announced positive Week 48 data from the Phase 2 open-label HELIOS clinical trial of AMX0035 (sodium phenylbutyrate [PB] and taurursodiol [TURSO, also known as ursodoxicoltaurine]) in adults living with Wolfram syndrome. These results were presented at the Joint Congress of the European Society for Pediatric Endocrinology (ESPE) and the European Society […]

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GABA and GLP-1 receptor agonist combination therapy modifies diabetes and Langerhans islet cytoarchitecture in a rat model of Wolfram syndrome

Publication: biomedcentral.com | Publication Date: 06 March 2025 Authors: Jagomäe, T., Velling, S., Tikva, T.B. et al. Abstract Wolfram syndrome (WS) is a rare autosomal disorder caused by WFS1 gene mutations, currently lacking approved treatments. Preclinical and clinical reports suggest that diabetes medications, such as glucagon-like peptide-1 receptor agonist (GLP1-RA), slow WS-related diabetes and neurodegeneration, […]

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6th Annual Golf Shootout, Sept 8, 2025

Join us for a great event on September 8, 2025 at Bogey Hills Country Club to support The Snow Foundation. The day will feature, 10 Par 3 holes with unique hole in on contests, 4 Par 4’s and 4 Par 5 Long Drive holes, a $100,000 Shootout for 10 Golfers. Download the flyer for registration […]

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