Registries

Disease registries can be used to collect real-world findings from people living with rare diseases, including disease signs and symptoms, measures of health and disease progression, clinical assessments, and treatment information. These findings are gathered over many years and can help to further our understanding about long-term disease progression and treatment effects, and to inform ongoing research. Click here to learn more about how registries accelerate disease research.

How Registries Help Advance Research

For people who are diagnosed with rare disease, they often have a feeling of isolation. They often have a feeling of being the only one who is responsible for explaining their disease to their own physicians.

 
So what a rare disease registry can provide is that it can provide a global community for each and every one of those patients. Rare disease registries are open structured studies and they can enroll individuals all over the world in order to share their stories and their experiences. It's really powerful for patients and physicians and scientists to be able to bring all of those experiences together and to create shared knowledge. In order to further understanding for the disease community, because registries run for many years, we can understand more about the long-term disease course and we can also understand more about the long-term effects of treatment. This can be extremely impactful for families, for clinicians and for the patient to anticipate and understand how that patient might do over time. A registry is much more than just a database. I think of the registry as the people. So first and foremost the patients, the patients who provide their invaluable data to become that global community. A registry is also the clinicians, the nurses, the genetic counselors, the physicians who are entering these data into the system. The registry is also the people who are asking the important research questions to be able to elucidate the questions that we don't know about a specific disease and to be able to meet those unmet medical needs for the patient community. Rare diseases by their very nature are extremely isolating for both patients and for clinicians. So one of the lasting impacts of the rare disease registries are to create the global community that didn't exist 30 years ago and there's a legacy there that will continue as we move forward and will continue on for years to come.

 


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