Blog

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 […]

My Wolfram Syndrome Journey – Pat Gibilisco – Pt. 1

Part I: The Beginning My journey started 28 years ago. My daughter was two years old when she was diagnosed with Type 1 Diabetes. We were able to manage it and still live a great life. When she was 12, she started to have other health issues, specifically, problems with vision and extreme thirst. Our […]

Jack Snow – His Cause Continues

Strong of Heart, Profiles of Notre Dame Athletes (written by Randy Covitz) Jack Snow’s daughter Stephanie pleaded and finally persuaded him to be in the birthing room when she was about to deliver her daughter Raquel. Tears filled Snow’s eyes when he held Raquel, his seventh grandchild, with the steady hands that caught 60 passes as […]

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 […]

Reflections

With 2016 behind us and the promise of 2017 ahead, I sit in my front room looking at the beautiful fire, wondering where the time has gone. My childhood was wonderful. I had two parents who loved me to the moon and back. My dad was one of a kind — handsome, charismatic and so […]

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 […]