Blog

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 […]

A British Consortium wins the joint call for proposals

Three British and one Australian research teams, coordinated by Professor Timothy Barrett, have been awarded the funding of 200,000 € by the Association syndrome de Wolfram, the Eye Hope Foundation and the Snow Foundation. Their project aims at “developing biomarkers that will show early evidence of benefit of a treatment; and patient self-report outcome measures […]

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 […]

Eye Problems in Wolfram Syndrome

Professor Patrick Yu-Wai-Man Affiliations John van Geest Centre for Brain Repair, Department of Clinical Neurosciences, University of Cambridge MRC Mitochondrial Biology Unit, University of Cambridge Cambridge Eye Unit, Addenbrooke’s Hospital, Cambridge University Hospitals Moorfields Eye Hospital and UCL Institute of Ophthalmology, London I am an eye doctor (ophthalmologist) with a particular interest in genetic eye […]

Washington University Wolfram Research Clinic completes its 8th consecutive year!

Dear Wolfram Families and Supporters: The Washington University Wolfram Research Clinic (or ‘natural history) study just finished its 8th consecutive year of assessing patients with Wolfram Syndrome. This work began in 2010, supported in part by the Snow Foundation. For the past 5 years, it has been supported by a National Institutes of Health grant […]

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 […]

Dr. Barrett meets with Wolfram Associations across the globe

Dear Friends and colleagues, I have been fortunate to visit the French Wolfram Association meeting in April in Paris; and the Spanish Wolfram association meeting in May in Almeria. I was humbled by the friendship and community spirit of these families; and they were very tolerant of my poor French and Spanish language skills. On […]

My Fight Continues – Pat Gibilisco – Pt. 3

Part 3: My Fight Continues – Pat Gibilisco Money was the only reason my child was not able to receive the drug that would potentially save her life.  The Snow Foundation, or I should say Stephanie Gebel, worked so hard to raise money for all children affected with WS.  She had to beg people, year […]

The Journey Continues – Pat Gibilisco – Pt. 2

Part 2: The Journey Continues Dr. Permutt and Jon Wasson, who helped discover the WS gene and named it WSF1, were leading the first-ever  WS clinic in St. Louis.  Dr. Hershey and many other fabulous doctors from Washington University in St. Louis and other areas of the United States, started the first database of WS […]