By Dr. Fumihiko Urano I received many questions about yesterday’s blog describing the clinical application of a new DNA sequence technology. So I would like to clarify a few things. 1. Cost There is a lot of discussion about the cost. Will our health insurance cover this? I believe that it will be covered in the […]
I have been thinking a lot about stem cell-based therapy for patients with Wolfram syndrome. We have created many induced pluripotent stem cell lines (iPS cell lines) using skin cells from patients with Wolfram syndrome. In theory, we can create any types of cells using these iPS cells. The idea of stem cell-based therapy for […]
We have the pleasure of sharing the posts from Dr. Urano’s blog “Combating Wolfram Syndrome”. Below are his posts for the week of July 21-25. Friday, July 25, 2014 Endoplasmic Reticulum Disease Clinic 1 Thank you for your feedback for my blog. http://wolframsyndrome.blogspot.com/2014/07/a-platform-for-providing-immediate-care_24.html We need to consider multiple factors and logistics for establishing “Endoplasmic Reticulum Disease […]
I presented my strategy for providing a cure for Wolfram syndrome at the clinic this weekend. I think there are three steps to achieve this. 1. Stop the progression2. Protect and Regrow remaining tissues3. Replace damaged tissues Our current focus is to “Stop the progression” of the disease. We are testing if FDA-approved drugs currently […]
I always appreciate the efforts of all the medical staff, administrative staff, volunteers, researchers, interpreters, and others who have been involved in the Wolfram syndrome research clinic. It is my privilege to serve for patients with Wolfram syndrome and work with dedicated people who have been involved in our Wolfram syndrome research. My team has […]
Wolfram research clinic is ongoing and our patients are going through many tests. At this year’s clinic, we are collecting blood samples from not only patients but also their parents and siblings. Why? The reason is we plan to measure “BIOMARKER” levels in these samples. What is a biomarker? A biomarker is a molecule found […]
By Dr. Fumihiko Urano Our annual Wolfram syndrome research clinic will start today, and I met with most of the patients and their families last night. I have been very impressed by them. In this clinic, we don’t provide any treatment. We just collect information and samples from patients, their parents and siblings. All of […]
Tuesday, July 8, 2014 By Dr. Fumihiko Urano Why are we creating eye cells using skin cells from patients? More accurately, we are creating two different types of retinal cells using stem cells derived from skin fibroblasts of patients with Wolfram syndrome. We can immediately use these cells to understand the mechanisms of the disease […]
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 […]
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 […]
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