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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.


  • Test efficacy of pantethine in PKAN cell and mouse models
  • Test efficacy and toxicity of compounds in PKAN cell culture and fly models
  • Analysis of Blood-Brain Barrier permeability of compounds
  • Rescue studies of most promising compounds in mouse
  • Pharmacokinetics and toxicity of most promising compound in mouse