Project Title: CoA-Z in Pantothenate Kinase-associated Neurodegeneration (PKAN)
Responsible Party: Penelope Hogarth, Oregon Health and Science University
The purpose of this study is to learn more about how people with the condition pantothenate kinase-associated neurodegeneration (PKAN) respond to a specialized study product. We are hoping to find out if the study product is safe, what effects—good and bad—the study product causes, and whether the study product changes certain measures of PKAN disease.
Project Title: NBIAready: Online Collection of Natural History Patient-reported Outcome Measures
Responsible Party: Susan Hayflick, Oregon Health and Science University
The purpose of this study is to learn more about Neurodegeneration with Brain Iron Accumulation (NBIA) Disorders. Data is being collected on three types of NBIA disorders: Pantothenate Kinase-Associated Neurodegeneration (PKAN), PLA2G6-associated Neurodegeneration (PLAN) and Beta-propeller Protein-associated Neurodegeneration (BPAN). The study will be (1) collecting information about how symptoms and findings in NBIA change over time and (2) identifying measures of NBIA that can be used in future clinical trials. Participants will follow links to a secure website every 6 months for a period of 5 years to electronically complete a set of rating scales as related to their NBIA disorder.