Clinical Trials Underway for PKAN

Clinical Trial

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.

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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.

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Best Practices for PKAN

PKAN Best Practices

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This article is also available on the Molecular Genetics and Metabolism website.

GeneReviews® - PKAN

Pantothenate Kinase-Associated Neurodegeneration

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More Information: PKAN

"Pantethine treatment is effective in recovering the disease phenotype induced by ketogenic diet in a pantothenate kinase-associated neurodegeneration mouse model". BRAIN, 2014
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"Intellectual and adaptive behaviour functioning in pantothenate kinase-associated neurodegeneration"
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"Metabolism & Energy Requirements in people with PKAN"
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