The NBIA Research Group at OHSU has developed a new five-year clinical study called PKANready. The purpose of this study is to help us better understand the natural history of PKAN, meaning how symptoms appear and change over time. By studying individuals with PKAN, we also hope to identify disease markers that can be used in future clinical trials. A disease marker is any symptom or measurement that happens reliably in a disease, changes predictably with disease progression, and becomes “better” with successful treatment. A disease marker could be an MRI finding, a protein level in the blood, or a rating scale to measure symptoms or function. Natural history studies provide data that serve as the foundation for future drug trials. In the future, we hope to replicate this five-year clinical study with other major forms of NBIA, such as PLAN, MPAN, BPAN, etc.
Do you have PKAN? Or maybe your son or daughter has PKAN? Then you can participate in our new online study from home!
Participation involves logging into 2 secure websites from home every 6 months for a period of 5 years. Each time you log in, you’ll be asked to complete a set of questions and rating scales that take about 15 minutes. Don’t worry if you forget to log in on time—we will remind you! We’ll also ask you to provide brief medical records confirming the PKAN diagnosis.
You may or may not benefit personally from participating in this study. As a participant you may contribute new information that may benefit patients in the future. To learn more and enroll in this online research study, click on the button below.
Susan Hayflick, Principal Investigator
NBIA Research Group, OHSU
3181 SW Sam Jackson Park Rd. L103
Portland, OR 97239
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