La Jolla Labs, USA Members of The Snow Foundation met with representatives from La Jolla Labs to investigate the role of RNA directed gene therapy, specifically AntiSense Oligonucleotides (ASOs), for the treatment of Wolfram syndrome. La Jolla Labs undertook an investigation to determine the potential for ASO application, specifically for the treatment of autosomal dominant […]
Principal Investigators- Drs. Raniero Chimienti and Giulio Frontino, Milan, Italy This work is done in collaboration with Telethon Foundation, Italy This project is designed to test whether immune cells that have been genetically corrected can help improve the inflammatory symptoms of Wolfram syndrome in a mouse model. By transplanting genetically corrected WS immune cells into […]
Extending into 2026 Principal Investigator-Felipe Chicani, MD, Sao Paolo, Brazil This patient comparison study will evaluate WS patients who have been treated with idebenone compared to an untreated control group to determine if idebenone can help slow the progression of WS symptoms, with a focus on vision loss. This project is currently in the planning […]
Principal investigator Dr. Cécile Delettre-Cribaillet, INM, Montpelier, France Extending into 2026: This work will be done in conjunction with a grant from the Be a Tiger Foundation This is an ongoing project evaluating and validating the potential for WFS1 wildtype gene transfer as atreatment for Wolfram syndrome. Preclinical data have already shown the ability of […]
Chief Medical and Scientific Officer From the Desk of the Dr. Sarah Gladstone, CMO/CSO As we enter 2026 and look back on 2025, it’s remarkable how much progress has been made in such a short time.Scientific research can be challenging—there are periods when progress feels slow and frustrating, and answers seem just out of reach. […]
January 31, 2026 Dr. Fumihiko Urano Dear Friends, I hope the new year has started well for you and your family. Thank you, as always, for being part of the Wolfram syndrome community. Your trust, patience, and partnership mean a great deal to us. Everything we do in the clinic, the clinical trial unit, and […]
Overview Professor Timothy Barrett presented updates on the Treat Wolfram trial, focusing on the use of sodium valproate for Wolfram syndrome. The trial, involving 42 valproate-treated and 21 placebo-treated patients, aimed to assess vision loss over 36 months. Despite a younger, healthier placebo group, valproate showed trends towards stabilizing HbA1c and insulin-adjusted HbA1c, and increasing […]
Overview Jamie Timmons from Amylyx presented the HELIOS trial results for Week 48, focusing on the investigational therapy AMX 35 (sodium phenylbutyrate and ursodil) for Wolfram syndrome. The trial, involving 12 participants, showed improvements in pancreatic beta cell function, insulin secretion, and glycemic control, with a significant reduction in hemoglobin A1c levels. The therapy was […]
Overview Vania Broccoli presented on Wolfram syndrome, highlighting the role of the WFS1 gene and its protein, Wolframin, in protein synthesis and folding. She discussed the impact of WFS1 mutations on retinal ganglion cells, leading to optic atrophy and vision loss. Broccoli’s research involved gene replacement therapy using adeno-associated viral vectors to restore Wolframin function […]
Overview Dr. Cécile Delettre-Criballet presented on therapeutic perspectives for Wolfram disease at a global virtual conference. Her team at the Institute for Neuroscience of Montpellier focuses on hereditary optic neuropathies and anterior segment diseases. They use genetic diagnostics, pathophysiological studies, and clinical trials to develop treatments. Key findings include the effectiveness of gene therapy in […]