For Patients
PKU Patient Registry
Welcome!
The PKU Patient Registry is an online registry for people with Phenylketonuria (PKU). It is sponsored by the National PKU Alliance and hosted by the National Organization for Rare Disorders (NORD®) on their IAMRARE® platform. This registry will collect information from individuals with PKU or their family members about the lived experience of PKU. The information gathered from the Registry can be used anonymously to influence PKU clinical management guidelines, shape NPKUA programs, and support PKU research initiatives, including clinical trials, studies for new therapeutic treatments, and management tools.
Have questions about the PKU Patient Registry? For a complete FAQ, please visit this page, which provides more information about the PKU Patient Registry. Some important highlights from the FAQ are included below.
What is a Patient Registry?
A patient registry is a collection of standardized information about a group of individuals who share a condition. The information may be used for a variety of purposes such as conducting natural history studies and supporting disease-specific clinical trial recruitment. The PKU Patient Registry serves to:
- Support the design of clinical trials that explore new rare disease treatments;
- Describe the people who have PKU and to better understand the variability and stages of PKU;
- Understand how PKU changes over a person’s lifetime;
- Learn about clinical practice patterns and variations over the course of treatment;
- Help to develop best practices, management guidelines, and recommendations so that clinicians can know how to give the best care to improve the quality of life and outcomes of people with PKU; and
- Identify people with PKU who might be willing to take part in other research studies or clinical trials. You will be able to choose whether you want to hear about these other studies.
What types of data will be collected in the PKU Patient Registry?
The PKU Patient Registry collects data on the following topics:
- Socio-demographics
- Medical history and diagnostics
- Treatment and disease progression
- Management of care
- Quality of life
- Genetic information
- Maternal PKU
Are the data secure?
The PKU Patient Registry follows strict government guidelines to assure patient information is protected. The platform is served over HTTPS, which means that the data are encrypted when being sent from the user’s browser to the NORD servers. The data are also kept encrypted in the NORD database. Communications between the registry platform application server and the database are also encrypted. As with any information one provides electronically, there is a very rare chance that privacy could be compromised. However, the registry and the security measures minimize the chance of this occurring.
Video links:
Janet Woodcock, Former Director CDER FDA on NORD Registry Program
Learn about the PKU Patient Registry