Work Package 5

Title of Work Package: Studies to define the potential of pantethine and pantethine derivatives to treat PKAN

Objective: WP5 will pursue preclinical work to develop a new and innovative therapeutic approach to treat PKAN. This approach is based on knowledge of the metabolic pathways in PKAN, and recent observation of rescue of the phenotype and the biochemical abnormalities in a PKAN model.

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