Figure 4.

Proposed alternative therapeutic strategies for PKAN. Disease-altering therapeutics for PKAN have thus far focused largely on metabolite supplementation or stimulation of CoA synthesis via PANK activation. Gene therapies based on adeno-associated viral vectors or other novel delivery mechanisms as well as inhibitors that target enzymes, such as PPP89, that counteract the flow of production of CoA from pantothenate are promising new approaches. PKAN, pantothenate kinase–associated neurodegeneration.