2014 Wolfram Research Clinic Update

Participants of the 2014 Wolfram Clinic
Participants of the 2014 Wolfram Clinic

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 and over 350 individual appointments!

We now have patients who have been seen up to 5 years in a row, providing an incredibly important view of the pattern of changes in symptoms that patients experience.

These data are now being analyzed so that we can determine the natural history of neurological changes in Wolfram Syndrome. We believe that neurological symptoms need to be targets of intervention and that we must have reliable markers of change that relate to meaningful outcome.  These are critical steps before any clinical trial can be initiated.

So far, our data indicate that there are very common, early neurologic features in Wolfram Syndrome, including reduced retinal thickness, color vision, smell identification, gait and balance and size and integrity of specific brain structures. Furthermore, we have early indications that some of these measures do not change reliably over a year’s time, whereas others may be more likely to change in a specific manner. This information could thus guide us in selecting the measures to monitor in any future clinical trial.

We hope to continue to collect data on our existing group of families, and add new families as we are able to get to these important answers more quickly and reliably.

Thank you to all of the families for participating, and to all of our colleagues and staff who helped make this unique and complicated clinic happen so smoothly.

Tamara Hershey, Ph.D.
Professor
Psychiatry, Neurology and Radiology
Washington University School of Medicine
Scientific Director of Clinic and
Principle Investigator, NIH R01 “Tracking Neurodegeneration in Early Wolfram Syndrome”

Bess Marshall, M.D.
Associate Professor
Pediatrics
Washington University School of Medicine
Medical Director of Clinic

Dr. Fumihiko Urano gave a presentation at the clinic on “A Cure for Wolfram Syndrome – 3 Steps”.  Click the link below to view the presentation.

2014 Wolfram Clinic Update