Research News

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 […]

2013 Wolfram Clinic

4th Annual Clinic on Wolfram Syndrome Held at St. Louis Children’s Hospital  July 17-20th The 2013 Wolfram Research Clinic was run over four days in mid July. A total of 22 patients, six of which were new, participated in MRI scans and many other measurements, totaling up to 326 individual appointments. This clinic is now truly international, with two families from […]