The Snow Foundation’s (TSF) mission since its inception has been to improve the lives of those affected by Wolfram Syndrome (WS) and lead the search for a cure. Historically, the outlook for individuals with WS was not good. When TSF was established eight years ago, virtually no clinical research was being done to develop better treatments for patients. Caregivers also faced a myriad of challenges with few places to turn for support.
Since its founding, TSF has proactively and strategically initiated, managed and funded research across the globe to accelerate the development of effective treatments, which we believe will ultimately lead to a cure for WS.
Despite long odds, the support, involvement, and generosity of our many friends and partners have fueled progress that is virtually unprecedented for a rare disease like WS. With your help, we have invested in research clinics, drug therapies, biomarkers and clinical trials that show promising signs of slowing the progression of this disease. These investments would not have been possible without your generosity.
TSF is now looking to expand its mission and lead a global movement of patients, families, doctors, and researchers who will work together to improve the odds for everyone affected by WS. Our new vision is bold: a world free of Wolfram Syndrome and other rare diseases.
We will accomplish this by not only expanding our investment in promising WS research, but also by launching critical programs and services that directly improve the lives of patients and their families. Examples of these investments include:
Research
Engage scientists worldwide to bring their expertise to bear on behalf of WS.
Eliminate many of the obstacles that once stood in the way of research, such as lack of tissue, disease models, and coordination among researchers.
Develop regenerative therapies to restore pancreas function (i.e., reverse diabetes), vision, hearing, and brain function.
Develop molecular therapies based on genome-editing technology for achieving a cure.
Apply powerful new technologies like CRISPR, single-cell sequencing, and DNA-encoded chemical libraries to the discovery of therapies that exploit WS’s unique vulnerabilities.
Drug Repurposing, thus accelerating the pace at which therapies developed for other neurodegenerative diseases can be tested in the lab and brought to clinical trials for WS patients.
Establish a patient navigation service that will help individuals facing WS overcome barriers to getting the best care possible – whether it’s finding the right doctors, understanding treatment options, or getting emotional support.
Develop educational materials, brochures, booklets and videos to help patients and families make informed medical decisions in order to get the best care possible.
Partner with companies and other nonprofit organizations to create resources and opportunities that address the needs of WS patients in all stages of life (childhood, adolescence, and adult).
Host an Annual Wolfram Syndrome Community Conference to provide opportunities for patients and their caregivers, clinicians and researchers to come together to learn about the latest advances in WS research and treatment, connect with one another and take action to achieve a shared vision for a better future for those affected by WS.
Set up consultation clinics to improve the clinical care of WS patients.
These exciting new endeavors will only take place through the direct support, insight and feedback of the entire WS community. To help begin and facilitate a conversation, I encourage you to complete this survey
and provide your thoughts on the programs and services that would be most beneficial to you. Your feedback is critical to our future success.
I am also pleased to announce that TSF will host the First Annual Wolfram Syndrome Community Conference on Saturday, July 13, 2019. This exciting conference will be held in conjunction with the annual Wolfram Syndrome Clinic at Washington University in St. Louis. It is our hope that participants—patients, caregivers, researchers and clinicians—come away from the first Community Conference more informed, connected to a supportive community, and empowered to more rapidly develop effective treatments and ultimately a cure for WS.
I firmly believe that a future in which everyone affected by WS is able to overcome the disease and maintain their quality of life is closer than we think. With the gift-giving season upon us, I hope you will consider joining me in support of our expanded mission by making a donation to TSF today.
Thank you so much for your continued support and generosity.
