Fosmetpantotenate (RE-024), a phosphopantothenate replacement therapy for pantothenate kinase-associated neurodegeneration: Mechanism of action and efficacy in nonclinical models
Table 8
Percentage of CoA derived from endogenous phosphopantothenic acid (unlabeled), fosmetpantotenate-derived pantothenic acid with rephosphorylation by WT PanK (+4 AMU), and fosmetpantotenate-derived phosphopantothenic acid (+6 AMU) following ICV administration of isotopically-labeled fosmetpantotenate to WT C57Bl6 micea.