Institute of Biomedicine and Translational Medicine, University of Tartu, Tartu, Estonia Professor Kaasik and his colleagues from the University of Tartu (Estonia) are currently searching for small molecular compounds to improve the function of endoplasmic reticulum that could have potential for the treatment of Wolfram Syndrome. They have identified several compounds with the ability to improve endoplasmic calcium homeostasis and neuronal […]
Washington University School of Medicine Drs Hershey, Marshall and the rest of the Wolfram Research Clinic team have been busy analyzing data that has already been collected and trying to plan for the next round of data collection during the current pandemic. We hope to start remote data collection (surveys, zoom interviews) this month, with […]
ULB Center for Diabetes Research, Brussels, Belgium GLP-1 analogs, such as liraglutide, exenatide and dulaglutide among others, are used to treat type 2 diabetes. These drugs are known to promote pancreatic beta cell function and survival, may cross the blood brain barrier, and might have potential beneficial effect on neurons and retinal cells. GLP-1 analogs […]
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, the protein responsible for Wolfram syndrome type 1. […]
Especialista en Medicina Familiar y Comunitaria Coordinadora del Equipo multidisciplinar del Síndrome de Wolfram Español Below are the most important activities led by Dr. Gema Esteban Bueno as head of the Spanish Clinical Wolfram Syndrome Team and scientific head of the Spanish Association for Research and Assistance to Wolfram Syndrome (AEIASW).. It also lists the scientific-bibliographic […]
University of Birmingham, United Kingdom
September 2020 Dear friends and colleagues, to recap, this is a trial of sodium valproate given by mouth, for 3 years, to try and slow down the progress of vision loss in Wolfram. It involves 4 countries in Europe, and is comparing the effects of sodium valproate to placebo. The trial recruited its first patient in […]
Wolfram syndrome is a rare genetic spectrum disorder characterized by insulin-dependent diabetes mellitus, optic nerve atrophy, and progressive neurodegeneration, and ranges from mild to severe clinical symptoms. There is currently no treatment to delay, halt, or reverse the progression of Wolfram syndrome, raising the urgency for innovative therapeutics for this disease. Here, we summarize our vision for developing novel treatment strategies and achieving a cure for Wolfram-syndrome-spectrum disorder.
Wolfram syndrome is a rare genetic spectrum disorder characterized by insulin-dependent diabetes mellitus, optic nerve atrophy, and progressive neurodegeneration, and ranges from mild to severe clinical symptoms. There is currently no treatment to delay, halt, or reverse the progression of Wolfram syndrome, raising the urgency for innovative therapeutics for this disease. Here, we summarize our vision for developing novel treatment strategies and achieving a cure for Wolfram-syndrome-spectrum disorder.
Dear Families: We (Drs. White, Marshall, Urano, and Hershey) are excited to announce that we are now funded to perform a clinical trial of liraglutide (Victoza®; NovoNordisk) in Wolfram Syndrome. Led by Drs. White and Marshall, this study will focus on the tolerability and safety of liraglutide and possible beneficial effects. We are inviting all […]
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