Work Package 4

Title of Work Package: A randomized, double-blind, placebo-controlled trial of deferiprone in patients with PKAN

Objective: The objective of WP4 is to establish the safety and efficacy of iron-chelating deferiprone in PKAN patients by conducting an adequately powered, randomized, double-blind, placebo-controlled trial. Study duration will be 18 months which will allow for assessment of symptomatic as well as disease-slowing neuroprotective effects of deferiprone as compared to the placebo group. (For more information, please visit



Data Safety Monitoring Board (DSMB) Memos regarding the safety of patients participation in the clinical trial:

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