TY  - GEN
AU  - Héron,Bénédicte
AU  - Valayannopoulos,Vassili
AU  - Baruteau,Julien
AU  - Chabrol,Brigitte
AU  - Ogier,Hélène
AU  - Latour,Philippe
AU  - Dobbelaere,Dries
AU  - Eyer,Didier
AU  - Labarthe,François
AU  - Maurey,Hélène
AU  - Cuisset,Jean-Marie
AU  - de Villemeur,Thierry Billette
AU  - Sedel,Frédéric
AU  - Vanier,Marie T
TI  - Miglustat therapy in the French cohort of paediatric patients with Niemann-Pick disease type C
SN  - 1750-1172
PY  - 2013///0103
KW  - 1-Deoxynojirimycin
KW  - administration & dosage
KW  - Adolescent
KW  - Age of Onset
KW  - Carrier Proteins
KW  - genetics
KW  - Child
KW  - Child, Preschool
KW  - Cohort Studies
KW  - Enzyme Inhibitors
KW  - Female
KW  - France
KW  - Glycoproteins
KW  - Humans
KW  - Infant
KW  - Intracellular Signaling Peptides and Proteins
KW  - Male
KW  - Membrane Glycoproteins
KW  - Niemann-Pick C1 Protein
KW  - Niemann-Pick Disease, Type C
KW  - drug therapy
KW  - Prospective Studies
KW  - Treatment Outcome
KW  - Vesicular Transport Proteins
N1  - Publication Type: Clinical Trial; Journal Article
UR  - https://doi.org/10.1186/1750-1172-7-36
ER  -