Washington University Wolfram Syndrome Research Clinic

Funding: National Institute of Child Health and Development (HD070855); 2012-2017
"Tracking Neurodegeneration in Early Wolfram Syndrome (TRACK)"
HRPO #201301004
Clinical Trials # (https://clinicaltrials.gov/)
Principal Investigator and Scientific Director: Tamara Hershey, PhD
Medical Director: Bess Marshall, MD

The purpose of the Washington University Wolfram Syndrome Research Clinic (WFS Research Clinic) is to better understand the development and progression of the neurological aspects of Wolfram syndrome, particularly during development. During annual research visits at WU and St. Louis Children’s Hospital, patients are seen by specialists in multiple disciplines (ophthalmology, neurology, urology, neuropsychology, genetics, physical therapy, psychiatry, sleep, audiology) and undergo magnetic resonance imaging(MRI) of the brain. In addition, Parents and patients are asked to fill out questionnaires. These data are critical in helping us understand the neurological issues in Wolfram Syndrome, and will directly inform the design and metrics used in future clinical trials.

Individuals with Wolfram Syndrome are needed for this research study. If you (your child) are interested in participating in the Wolfram Syndrome Research Clinic please contact Samantha Ranck, MSW at (314) 362-6514 or rancks@npg.wustl.edu. You will be asked to complete an on-line screening questionnaire to determine your eligibility.

Clinical Care: In addition to serving as the Medical Director of the WFS clinic, Dr. Marshall is a pediatric endocrinologist who directs a Clinical Center of Excellence for Wolfram Syndrome patients at Washington University School of Medicine (Bess Marshall, M.D.; marshall@kids.wustl.edu or 314-454-6051). Patients with Wolfram Syndrome interested in being seen by Dr. Marshall can contact her directly.

Wolfram Syndrome Clinical Guidelines: http://www.euro-wabb.org/images/euro-wabb/guidelines/Wolfram_guideline_V14_%2028_04_2014.pdf

Additional Research Opportunities:

University of Maryland Brain and Tissue Bank.
The National Institute of Child Health and Human Development (NICHD) supports the Brain and Tissue Bank for Developmental Disabilities (http://medschool.umaryland.edu/btbank/family/).

Washington University Wolfram Syndrome Research Study Group

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Raise and Join Your Hands for Rare Disease Day 2018
The "Wolfram" clinic staff at Washington University in St. Louis