Entries by Fumihiko Urano

Dr. Urano’s November Update

November 4, 2017 Dear Friends, It has been a while since we last talked. I hope you are enjoying this Fall season with your family and friends. I think about the value of family and friends a lot lately. Support from family and friends keeps me going. So I appreciate your continued support, encouragement, and […]

Dr. Urano’s September update

September 22, 2017 Dear Friends, It is nice to “meet” you again. Thank you for your generous and continued support for our therapeutic development for Wolfram syndrome. With the support from the Snow Foundation, multiple patient organizations and supporters around the world, and federal grants, I could maintain the Wolfram syndrome program to study the […]

Dr. Urano’s August update

Dear Friends, I hope you had a wonderful summer season. I would like to update you about our progress. Our clinical trial of dantrolene sodium in patients with Wolfram syndrome is ongoing. We have been monitoring the safety, tolerability, and efficacy of the drug. Some patients have been taking dantrolene sodium for more than 6 […]

Dr. Urano’s June Wolfram Research Update

Dear Friends, It’s a spectacular Saturday morning in Saint Louis as I write this to you. I always appreciate your continued support, encouragement, and kind words. I have received many questions regarding our ongoing clinical trial, as well as questions related to our next step lately. Although we don’t have a concrete plan yet, we […]

Clinical Trial Update by Fumihiko Urano, M.D.

Dear Friends, I would like to update you about the status of our clinical trial of dantrolene sodium in patients with Wolfram syndrome. Dantrolene is a US Food and Drug Administration (FDA)-approved drug currently used for different medical conditions. Based on our data, we received orphan drug designation of dantrolene sodium for the treatment of […]

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 […]

3 Things on my Mind When I Work on Wolfram syndrome

While working on Wolfram syndrome there are three things that are always on my mind: improving clinical care, raising awareness, and providing a cure. These three things motivate myself and my team to work hard every day.   Improving Clinical Care Our effort to understand the progression and neurological symptoms of Wolfram syndrome will help […]

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 […]

High sugar levels in the brain and neurodegeneration

My top priorities are to: 1) repurpose a FDA-approved drug and 2) develop novel drugs for Wolfram syndrome. I am also looking into environmental factors, diets, and life-styles that can potentially delay the progression of Wolfram syndrome. One of the possibilities I am looking into is to keep our blood sugar steady (i.e., maintain normoglycemia). […]