Clinical Care Update
Washington University School of Medicine
Clinical Care Update- Bess Marshall, MD
Dear Wolfram families,
As you now have heard from Samantha, I have determined that the Wolfram Research Clinic that was tentatively All of the Wolfram team is sad that we will not be able to see all of you in July. Please know that you are still a very high priority and that this bump will not derail the work at Washington University. We will not allow that to happen! You likely all saw the update from Dr. Barrett in the UK that his intervention trial is not yet underway as they also work through issues, but that it is making progress. Dr. Urano’s dantrolene study is moving along and he will be updating you on those results soon.
The Association du Syndrome de Wolfram meeting is coming up in June and Drs. Hershey and Urano and I will be going to hear updates from the other groups working on the syndrome alongside us, so we will update you in the next newsletter.
Some of the information you all have contributed by participating in the TRACK study was used to develop a paper led by Dr. Barrett’s group: Monogenic diabetes syndromes: Locus-specific databases for Alstrom, Wolfram, and Thiamine-responsive megaloblastic anemia. Human Mutation. 38(7):764-777, 2017 Jul.
This paper analyzes the specific gene changes in 309 people with WFS1 gene alterations in order to determine which changes are likely to cause a particular presentation in a person – for example, some genetic changes cause full-blown Wolfram Syndrome, which others cause diabetes mellitus without other features, others cause hearing loss without other features, etc. This will be very helpful information for patients at the time of diagnosis, getting their genetic testing results and wondering what to expect for their health.
As always, please get in touch if you need assistance with your health or with letters to insurance, etc.
All the best,
Bess Marshall, MD
Pediatric Endocrinologist
Medical Director, WU Wolfram Syndrome Research Clinic
Washington University School of Medicine
Email: Marshall@kids.wustl.edu
Need Help? For questions or requests regarding the Wolfram Syndrome Research Clinic please contact the WFS Research Clinic Coord., Samantha Ranck, MSW at 314.362.6514 or rancks@npg.wustl.edu