A therapeutic approach to pantothenate kinase associated neurodegeneration: a pilot study

Abstract Background Neurodegeneration with brain iron accumulation (NBIA) is a group of genetic neurological disorders frequently associated with iron accumulation in the basal nuclei of the brain characterized by progressive spasticity, dystonia, muscle rigidity, neuropsychiatric symptoms, and reti...

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Main Authors: Alessandra Pereira, Carolina Fischinger Moura de Souza, Mónica Álvarez-Córdoba, Diana Reche-López, José Antonio Sánchez-Alcázar
Format: Article
Language:English
Published: BMC 2024-11-01
Series:Orphanet Journal of Rare Diseases
Online Access:https://doi.org/10.1186/s13023-024-03453-x
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