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Miglustat in Niemann-Pick disease type C patients: a review

OBJECTIVE: Niemann-Pick disease type C (NP-C) is a rare, autosomal recessive, neurodegenerative disease associated with a wide variety of progressive neurological manifestations. Miglustat is indicated for the treatment of progressive neurological manifestations in both adults and children. Since ap...

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Detalles Bibliográficos
Autores principales:	Pineda, Mercè, Walterfang, Mark, Patterson, Marc C.
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	BioMed Central 2018
Materias:	Review
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6094874/ https://www.ncbi.nlm.nih.gov/pubmed/30111334 http://dx.doi.org/10.1186/s13023-018-0844-0

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