Miglustat therapy in the French cohort of paediatric patients with Niemann-Pick disease type C.
Héron, Bénédicte
Miglustat therapy in the French cohort of paediatric patients with Niemann-Pick disease type C. [electronic resource] - Orphanet journal of rare diseases Jun 2012 - 36 p. digital
Publication Type: Clinical Trial; Journal Article
1750-1172
10.1186/1750-1172-7-36 doi
1-Deoxynojirimycin--administration & dosage
Adolescent
Age of Onset
Carrier Proteins--genetics
Child
Child, Preschool
Cohort Studies
Enzyme Inhibitors--administration & dosage
Female
France
Glycoproteins--genetics
Humans
Infant
Intracellular Signaling Peptides and Proteins
Male
Membrane Glycoproteins--genetics
Niemann-Pick C1 Protein
Niemann-Pick Disease, Type C--drug therapy
Prospective Studies
Treatment Outcome
Vesicular Transport Proteins
Miglustat therapy in the French cohort of paediatric patients with Niemann-Pick disease type C. [electronic resource] - Orphanet journal of rare diseases Jun 2012 - 36 p. digital
Publication Type: Clinical Trial; Journal Article
1750-1172
10.1186/1750-1172-7-36 doi
1-Deoxynojirimycin--administration & dosage
Adolescent
Age of Onset
Carrier Proteins--genetics
Child
Child, Preschool
Cohort Studies
Enzyme Inhibitors--administration & dosage
Female
France
Glycoproteins--genetics
Humans
Infant
Intracellular Signaling Peptides and Proteins
Male
Membrane Glycoproteins--genetics
Niemann-Pick C1 Protein
Niemann-Pick Disease, Type C--drug therapy
Prospective Studies
Treatment Outcome
Vesicular Transport Proteins