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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Detalhes bibliográficos
Publicado no:J Neurol
Main Authors: Sedel, Frédéric, Chabrol, Brigitte, Audoin, Bertrand, Kaphan, Elsa, Tranchant, Christine, Burzykowski, Tomasz, Tourbah, Ayman, Vanier, Marie T., Galanaud, Damien
Formato: Artigo
Idioma:Inglês
Publicado em: Springer Berlin Heidelberg 2016
Assuntos:
Original Communication
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC4859844/
https://ncbi.nlm.nih.gov/pubmed/26984608
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1007/s00415-016-8051-1
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