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Long‐term survival outcomes of patients with Niemann‐Pick disease type C receiving miglustat treatment: A large retrospective observational study

Miglustat has been indicated for the treatment of Niemann‐Pick disease type C (NP‐C) since 2009. The aim of this observational study was to assess the effect of miglustat on long‐term survival of patients with NP‐C. Data for 789 patients from five large national cohorts and from the NPC Registry wer...

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Detalles Bibliográficos
Autores principales:	Patterson, Marc C., Garver, William S., Giugliani, Robert, Imrie, Jackie, Jahnova, Helena, Meaney, F John, Nadjar, Yann, Vanier, Marie T., Moneuse, Patrick, Morand, Olivier, Rosenberg, Daniel, Schwierin, Barbara, Héron, Benedicte
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	John Wiley & Sons, Inc. 2020
Materias:	Original Articles
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7540716/ https://www.ncbi.nlm.nih.gov/pubmed/32324281 http://dx.doi.org/10.1002/jimd.12245

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