Research News

2012 Wolfram Syndrome Research Update

Compiled By: Tamara Hershey, Ph.D., Bess Marshall, M.D., Fumi Urano, M.D., Ph.D. and the WU Wolfram Study Group members Overview This past year has been an exciting but also difficult one for our group and for the Wolfram community. While we have made great gains in our research efforts, we also lost our friend, colleague […]

Treatment target for diabetes, Wolfram syndrome

August 7, 2012 Inflammation and cell stress play important roles in the death of insulin-secreting cells and are major factors in diabetes. Cell stress also plays a role in Wolfram syndrome, a rare, genetic disorder that afflicts children with many symptoms, including juvenile-onset diabetes. The bright green spots are TXNIP molecules, potential treatment targets for […]

Wolfram Syndrome Researcher Makes Headlines

Tamara Hershey was recently featured on the Washington University School of Medicine website for her studies in how fluctuations in glucose levels can influence the brain .  As well as her work as a neuroscientist, she devotes much of her time studying Wolfram syndrome. “Endocrinologists usually are interested in Wolfram syndrome because it’s a genetic form […]

Snow White Fellowship

Wolfram Moms Join Forces to Create “Snow White Fellowship” In the fall of 2013, Stephanie Snow Gebel and Beth White decided to join forces and create “The Snow White Fellowship” at Washington University School of Medicine. The Jack & J.T. Snow Scientific Research Foundation and The Ellie White Foundation for Rare Genetic Disorders decided to join […]

Patient Based Therapeutics – New Drug Candidates

New Drug Candidates As I mentioned in my previous blogs, we have identified three FDA-approved drugs, one supplement, and new groups of drugs that can potentially delay the progression of Wolfram syndrome. We have been testing the efficacy of these drugs in cells from patients and two animal models of Wolfram syndrome. Preliminary data look […]

Patient-Based Therapeutics Part 6

Wolfram Syndrome iPS Cells Progress I received many emails regarding our progress on Wolfram syndrome induced pluripotent stem cells (iPS cells) in the past two weeks. I would like to update you on a few things. As I mentioned in my previous blogs, we have created many iPS cells from skin cells of patients with […]

Patient Based Therapeutics – Part 4

Patient-Based Therapeutics Part 4 – Drug Screening Progress Based on the data obtained from our patients, animal models, and cell models of Wolfram syndrome, we found that calcium depletion of the endoplasmic reticulum (ER) plays a role in the pathogenesis of Wolfram syndrome. So we have been looking for drugs that can prevent ER calcium-depletion-mediated […]

Wolfram Clinic

In September of 2009 we established the Internet-based International Wolfram Syndrome Registry. As the prevalence is estimated at 1 in 200,000-700,000, the Registry was established to provide a cohort of patients for future studies. We have extended the clinical arm of this project to acquire longitudinal data on the pattern of disease progression and identify […]

Research Reports From Washington University

Introducing… the Washington University Wolfram Syndrome Study Group! By Dr. Tamara Hershey I would like to tell you about the big picture of research and clinical activities at Washington University focused on Wolfram Syndrome. There are three parts to this effort 1) Diagnostic markers and treatment  development for Wolfram syndrome using animal models and human […]

Patient-Based Therapeutics Part 2

Research Update from Dr. Fumihiko Urano Patient-Based Therapeutics Part 2 We are taking an unconventional approach to develop therapeutics for Wolfram syndrome. I would call it “patient-based therapeutics.” This implies a few things. One of these is the “mechanism-based treatment.” How can we achieve this component of “patient-based therapeutics” for Wolfram syndrome? Here are our […]