DID YOU KNOW? The two types of Wolfram syndrome (type 1 and type 2) are primarily differentiated by their genetic cause. Variations (mutations) in the WFS1 gene are responsible for about 90% of Wolfram syndrome type 1 cases. This gene encodes wolframin, a protein that is important for the proper functioning of the endoplasmic reticulum (the part of a cell that is involved in […]
WASHINGTON UNIVERSITY’S 2019 WOLFRAM RESEARCH CLINIC UPDATE Wait no more – the 2019 WU Wolfram Research Clinic planning is underway! We’re sure many of you have lots of questions and hopefully some of them will be answered here. If not, you can always contact Samantha directly. Her contact information is below. Due to the number […]
As discussed in a previous newsletter, we have learned that anxiety and depression symptoms are fairly common in people with Wolfram Syndrome. These symptoms are also very common in people without Wolfram Syndrome, and may be influenced by genes, life stress, and other factors. In preliminary examination of our data, I have also noticed some […]
Washington University School of Medicine Dear Wolfram Community: I am very pleased to announce that we have received notification from the NIH that our Wolfram natural history study has been OFFICIALLY FUNDED! This means that we can start having our Wolfram Research Clinics again, probably starting next summer and continuing for 5 more years! Samantha, […]
Our team’s goal is to discover, test and develop treatments in order to prevent or limit visual impairment and to improve the autonomy and the quality of life of patients. Our efforts focus on a very severe form of syndromic Inherited Optic Neuropathy: Wolfram Syndrome (WS). WS is characterized by a rapid degeneration of retinal […]
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.
Washington University School of Medicine Clinical Care Update- Bess Marshall, MD Dear Wolfram families, As you now have heard from Samantha, I have determined that the Wolfram Research Clinic that was tentatively All of the Wolfram team is sad that we will not be able to see all of you in July. Please know […]
Washington University School of Medicine Wolfram Research Clinic Update- Tamara Hershey, PhD Dear Research Clinic Families, As you now have heard from Samantha, I have determined that the Wolfram Research Clinic that was tentatively scheduled for July 2018 will not be able to happen. This was a very difficult decision, but ultimately, we felt […]
1st teleconference meeting report Tuesday 24 April, 2018 Report written by: Virginie Picard (Association du syndrome de Wolfram) Report validated by: prof. Timothy Barrett, project coordinator The first update teleconference meeting on Biomarker project led by professor Tim Barrett took place on Tuesday 24 April, six months after the official beginning of the project. Representatives […]
Mitochon Pharmaceuticals, Inc., Blue Bell, Pennsylvania, is sponsoring a Proof of Concept (POC) study in Wolfram mice using a mitochondrial target approach to attenuate diabetes, behavioral and functional decline. Mitochon has developed clinical stage (Phase I ready) pharmaceuticals that modulate mitochondrial physiology. These compounds, MP101 and MP201, have shown merit in animal models of vision […]
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