Dr. Giulio Frontino and Dr. Maurizio Delvecchio discuss crafting national guidelines for the diagnosis and care of individuals with Williams Syndrome. You can listen to their presentation by following the link provided: https://youtu.be/PssLjWZgsFE
Dr. Fumihiko Urano Washington University School of Medicine, USA Dear Friends, I hope you had a wonderful summer. As the number of patients referred to us continues to grow, our team feels an even greater urgency to develop safe and effective treatments for Wolfram syndrome. I’m pleased to share the latest updates with you: Ongoing […]
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Dr. Cecile Delettre Neuroscience Institute of Montpellier, France Recently we have identified a family of molecules with the capacity to stimulate significantly the growth of retinal ganglion cells in vitro in a model of optic atrophy. We developed a zebrafish model with an optic atrophy and we have treated these fishes with one of these […]
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 […]
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