The registry is not only important to current patients but is important because it will impact patients for generations. Our researchers and our donors are creating a legacy. We have been very fortunate to meet the financial needs of the study so far but we cannot become complacent considering the amount need to fund the remainder of the project.
There is now a dedicated phone number for all patients to call from anywhere to enter the patient registry. It is answered at the first registry site at Yale and they will make arrangements to get you to a location most convenient for you. Here is a dedicated line for people to call to enter the Patient Registry: (203)376-6043 or email ayse.coskun@yale.edu.
A Deeper Understanding of the Patient Registry
The registry is not only important to current patients but is important because it will impact patients for generations.
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 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 medications. 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.
We would like to take this opportunity introduce Dr. Ayse Coskun, our new study lead coordinator at Yale, to the Wilson Disease Association membership. Ayse joined us in May 2019 and is working with each of our study sites to help initiate their active patient recruitment. We are pleased that recruitment has already started at Baylor College of Medicine in Houston, Texas where Dr. Sanjiv Harpavat has continued the work initiated by Dr. Tamir Miloh. Already three patients were recruited into the study at Baylor, and more will follow. Soon to begin enrollment this Summer are Florida Hospital, led by Dr. Regy Gonzalez, Seattle Childrens under the direction of Dr. Sihoun Hahn, and the Royal Surrey and Kings College Hospitals in the UK where Drs. Aftab Ala and Michelle Camarata are lead investigators.
