Fosmetpantotenate (RE-024), a phosphopantothenate replacement therapy for pantothenate kinase-associated neurodegeneration: Mechanism of action and efficacy in nonclinical models

In cells, phosphorylation of pantothenic acid to generate phosphopantothenic acid by the pantothenate kinase enzymes is the first step in coenzyme A synthesis. Pantothenate kinase 2, the isoform localized in neuronal cell mitochondria, is dysfunctional in patients with pantothenate kinase-associated...

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Detalles Bibliográficos
Autores principales: Elbaum, Daniel, Beconi, Maria G., Monteagudo, Edith, Di Marco, Annalise, Quinton, Maria S., Lyons, Kathryn A., Vaino, Andrew, Harper, Steven
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Public Library of Science 2018
Materias:
Research Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5844530/
https://www.ncbi.nlm.nih.gov/pubmed/29522513
http://dx.doi.org/10.1371/journal.pone.0192028

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