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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:
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