C. Ben Mamoun

A Selective PanK3 Activator Restores Coenzyme A in Cellular and Mouse Models of Pantothenate Kinase-Associated Neurodegeneration.

Pantothenate kinase-associated neurodegeneration, PKAN, is a rare progressive neurodegenerative disorder associated with iron accumulation in the brain. The disease, which causes early immobility and often death by early adulthood, is caused by loss of function mutations in the human PANK2 gene, encoding a key enzyme i...