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Normalisation of brain spectroscopy findings in Niemann–Pick disease type C patients treated with miglustat

Niemann–Pick disease type C (NP-C) is a fatal progressive neurolipidosis involving neuronal storage of cholesterol and gangliosides. Miglustat, an inhibitor of glycosphingolipid synthesis, has been approved to treat neurological manifestations in adults and children with NP-C. This open-label observ...

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Detalles Bibliográficos
Autores principales:	Sedel, Frédéric, Chabrol, Brigitte, Audoin, Bertrand, Kaphan, Elsa, Tranchant, Christine, Burzykowski, Tomasz, Tourbah, Ayman, Vanier, Marie T., Galanaud, Damien
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	Springer Berlin Heidelberg 2016
Materias:	Original Communication
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4859844/ https://www.ncbi.nlm.nih.gov/pubmed/26984608 http://dx.doi.org/10.1007/s00415-016-8051-1

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