Miglustat therapy in the French cohort of paediatric patients with Niemann-Pick disease type C-Reference-Cited by-同舟云学术

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

Published:2012 Issue:1 Volume:7 Page:36
ISSN:1750-1172
Container-title:Orphanet Journal of Rare Diseases
language:en
Short-container-title:Orphanet Journal of Rare Diseases

Author:

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

Publisher

Springer Science and Business Media LLC

Subject

Pharmacology (medical),Genetics (clinical),General Medicine

Reference27 articles.

1. Niemann-Pick disease type C

2. Recommendations on the diagnosis and management of Niemann-Pick disease type C

3. Natural history of Niemann-Pick disease type C in a multicentre observational retrospective cohort study

4. Miglustat in patients with Niemann-Pick disease Type C (NP-C): A multicenter observational retrospective cohort study

5. Niemann-Pick disease type C

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