Miglustat therapy in the French cohort of paediatric patients with Niemann-Pick disease type C

BACKGROUND: Niemann-Pick disease type C (NP-C) is a rare neurovisceral lysosomal lipid storage disease characterized by progressive neurological deterioration. Published data on the use of miglustat in paediatric patients in clinical practice settings are limited. We report findings from a prospecti...

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Autores principales: Héron, Bénédicte, Valayannopoulos, Vassili, Baruteau, Julien, Chabrol, Brigitte, Ogier, Hélène, Latour, Philippe, Dobbelaere, Dries, Eyer, Didier, Labarthe, François, Maurey, Hélène, Cuisset, Jean-Marie, de Villemeur, Thierry Billette, Sedel, Frédéric, Vanier, Marie T
Formato: Artigo
Lenguaje:Inglês
Publicado: BioMed Central 2012
Materias:
Research
Acceso en línea:https://ncbi.nlm.nih.gov/pmc/articles/PMC3465012/
https://ncbi.nlm.nih.gov/pubmed/22676771
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1186/1750-1172-7-36
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