Wilson Disease Association
Tax ID: 16-1154397
BASED: Milwaukee, WI, United States
CHARITY WEBSITE: www.wilsonsdisease.org
Wilson Disease Association
End Wilson Disease
The Wilson Disease Association funds research and facilitates and promotes the identification, education, treatment, and support of patients and other individuals affected by Wilson disease. The Wilson Disease Association was pleased to announce the kickoff for the patient registry in 2017. What is a patient registry?
A patient registry collects information about patients who are affected by a particular condition. Patient registries and databases are key instruments to develop clinical research in the field of rare diseases (RD) and improve patient care as well as healthcare planning and policy. They are the only way to gather data from many sources to achieve a sufficient sample size and quality of information for epidemiological and clinical research. Therefore patient registries are vital to identifying important issues that need to be addressed by clinical trials and to facilitate their planning and recruitment of patients.
Why a patient registry for Wilson Disease?
Though treatments have been available for over 60 years for Wilson disease (WD), unmet needs and unanswered questions remain about diagnosis, best therapy, individualization of treatment, and the monitoring of therapy. There is currently no multicenter registry for WD in the US. In conjunction with leading Wilson Disease clinicians, we are in the process of developing a patient registry for WD at Yale University that will be expanded to multiple sites in the US and at one site in the UK. The registry will collect over a minimum period of 5 years very detailed data on new and treated WD patients. In addition to the data registry, a bio-bank for specimens and DNA from WD patients will also be established.
The information obtained from this project will help physicians and researchers determine the best tests for diagnosing WD, the ideal way to treat patients and how best to monitor therapy. They will also learn more about the frequency of side effects of current treatments. Results will improve the definitions of successful treatment, failure of therapy and the reasons for considering alternative therapy. This data will also be useful for the development of new therapies and comparisons between available treatments. This will have a direct impact on improving the future health and quality of life of patients with WD.
The Wilson Disease Association (WDA) is not aware of any other multicenter, multinational Wilson Disease registry with prospective data collection in the world. The results of this research will benefit current and future WD patients globally.
The multicenter registry research project will take place at several of the WDA’s designated Centers of Excellence. Yale University will begin recruiting patients to participate beginning October 2017. Within the following months, recruitment is planned to begin at the University of Michigan followed by Baylor University. In the following year we
plan recruitment to begin at the University of Surrey in the United Kingdom and at the University of Washington in Seattle, and next in Florida. The project aims to recruit 300 patients over a five year period.
We are excited to announce the formal kick-off of raising $1,000,000 for this important research project. We are accepting donations designated for “registry research” and our annual Big Wow events have been dedicated to
The Wilson Disease Association was pleased to announce the kickoff for the patient registry in 2017.
What is a patient registry?