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Population Pharmacokinetic Model of N-acetylmannosamine (ManNAc) and N-acetylneuraminic acid (Neu5Ac) in Subjects with GNE Myopathy

BACKGROUND: GNE myopathy is a rare genetic muscle disease resulting from deficiency in an enzyme critical for the biosynthesis of N-acetylneuraminic acid (Neu5Ac, sialic acid). The uncharged Neu5Ac precursor, N-acetylmannosamine (ManNAc), is under development as an orphan drug for treating GNE myopa...

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Detalles Bibliográficos
Autores principales:	Van Wart, Scott, Mager, Donald E., Bednasz, Cindy J., Huizing, Marjan, Carrillo, Nuria
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	Springer International Publishing 2021
Materias:	Original Research Article
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8206310/ https://www.ncbi.nlm.nih.gov/pubmed/33893973 http://dx.doi.org/10.1007/s40268-021-00343-6

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