Research News

End Of The Year Update

Dear Friends, My highest priority right now is to work out the logistics for the upcoming clinical trial. As our medical center announced in November this year, we plan to start a phase 1b safety clinical trial of dantrolene sodium in patients with Wolfram syndrome. Dantrolene sodium is an FDA-approved drug utilized for the treatment […]

Researchers launch first clinical trial for Wolfram syndrome

  Researchers at Washington University School of Medicine in St. Louis are launching a new clinical trial to assess the safety of a drug treatment for patients with the rare disease Wolfram syndrome. Wolfram syndrome affects about one in every 500,000 people worldwide. Many of those patients die prematurely from the disease. Patients with Wolfram […]

7th Annual Washington University Wolfram Syndrome Research Clinic Meeting

2015 Wolfram Research Clinic Update

Three Drug Targets. What is the difference?

We have identified three drug targets for Wolfram syndrome in the past two years. These are MANF, Calpain 2, and IRE1. What is the difference between these three molecules. Here is a short summary. MANF: Enhances survival and proliferation of remaining cells. Caplain 2: Activates cells death signal in Wolfram and Diabetes. So we need […]

The “Mini-Clinic”: News from the Wolfram Research Clinic at Washington University

Last summer, we had a successful research clinic, with 24 patients and their families attending from all over the world and the US. We welcomed three new patients to our group this year and in a whirlwind 4 days, we performed 22 MRI scans and over 350 individual appointments! Since then, we have identified additional […]

Stem cell-based therapy Q & A – Medical Use

Here are two other questions I often get. Q: How can we use stem cells for the treatment of Wolfram syndrome and Type 1 diabetes. A: Stem cells can differentiate into specific cell types including insulin-producing cells, retinal cells, and brain cells.  The most important function of stem cells, especially induced pluripotent stem cells (iPS cells), […]

2014 Wolfram Research Clinic Update

Updates from the 2014 Wolfram Research Clinic at Washington University We had an exceptionally successful research clinic this year, with 24 patients and their families attending from all over the world and the US. We welcomed three new patients to our group this year and in a whirlwind 4 days, we performed 22 MRI scans […]

iPS cells and Personalized Medicine

We have created many induced pluripotent stem cells (iPS cells) from skin cells of Wolfram syndrome patients. Because these cells can be differentiated into any types of cells, including brain cells, eye cells, and insulin-producing pancreatic cells, we can use these cells to replace damaged tissues in our patients in the future. In addition to […]

The article I read over and over

I have read many articles related to Wolfram syndrome. The most-read article is entitled “Neurodegeneration and diabetes: UK nationwide study of Wolfram (DIDMOAD) syndrome” describing the natural history of Wolfram syndrome. This article was written by Timothy Barrett, MB, BS (MD), PhD in 1995.http://www.ncbi.nlm.nih.gov/pubmed/7490992 I read this paper over and over when I started working on Wolfram syndrome […]