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. 2022 Jan 15;298(3):101577. doi: 10.1016/j.jbc.2022.101577

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© 2022 The Authors

This is an open access article under the CC BY license (http://creativecommons.org/licenses/by/4.0/).

PMC Copyright notice

Current stage of development for key PKAN therapeutics. Some repurposed drugs focused on iron chelation (trial ID: NCT01741532) and metabolite supplementation (trial ID: NCT03041116) have completed phase III clinical trials in PKAN patients with little to no success. These drugs have been discontinued as treatments for PKAN. 4′-phosphopantotheine supplementation (Coa-Z trial ID: NCT04182763), acetyl-4′-phosphopantotheine supplementation, and PANK3 activation by BBP671 are promising new strategies for PKAN treatment at various stages of clinical evaluation. PKAN, pantothenate kinase–associated neurodegeneration.