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

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

<p><b>Abstract</b></p> <p><b>Background</b></p> <p>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 paedi...

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Bibliographic Details
Main Authors: 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, Sedel Frédéric, Vanier Marie T
Format: Article
Language:English
Published: BMC 2012-06-01
Series:Orphanet Journal of Rare Diseases
Subjects:
Niemann-Pick disease type C
Paediatric
Miglustat
Online Access:http://www.ojrd.com/content/7/1/36

Internet

http://www.ojrd.com/content/7/1/36

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