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  2. Details for: Miglustat therapy in the French cohort of paediatric patients with Niemann-Pick disease type C.
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Miglustat therapy in the French cohort of paediatric patients with Niemann-Pick disease type C. [electronic resource]

By:
  • Héron, Bénédicte
Contributor(s):
  • 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
Producer: 20130103Description: 36 p. digitalISSN:
  • 1750-1172
Subject(s):
  • 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
Online resources:
  • Available from publisher's website
In: Orphanet journal of rare diseases vol. 7
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Publication Type: Clinical Trial; Journal Article

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

APA

Héron B., Valayannopoulos V., Baruteau J., Chabrol B., Ogier H., Latour P., Dobbelaere D., Eyer D., Labarthe F., Maurey H., Cuisset J., de Villemeur T. B., Sedel F. & Vanier M. T. (20130103). Miglustat therapy in the French cohort of paediatric patients with Niemann-Pick disease type C. : Orphanet journal of rare diseases.

Chicago

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 and Vanier Marie T. 20130103. Miglustat therapy in the French cohort of paediatric patients with Niemann-Pick disease type C. : Orphanet journal of rare diseases.

Harvard

Héron B., Valayannopoulos V., Baruteau J., Chabrol B., Ogier H., Latour P., Dobbelaere D., Eyer D., Labarthe F., Maurey H., Cuisset J., de Villemeur T. B., Sedel F. and Vanier M. T. (20130103). Miglustat therapy in the French cohort of paediatric patients with Niemann-Pick disease type C. : Orphanet journal of rare diseases.

MLA

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 and Vanier Marie T. Miglustat therapy in the French cohort of paediatric patients with Niemann-Pick disease type C. : Orphanet journal of rare diseases. 20130103.

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