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Substrate reduction therapy with miglustat for type 1 Gaucher disease: A retrospective analysis from a single institution

INTRODUCTION. Gaucher disease (GD) is an infrequent progressive multisystem lysosomal storage disorder caused by the deficient activity of the lysosomal enzyme, glucocerebrosidase. A retrospective, single-center analysis of the clinical experience concerning the use of miglustat (N-butyldeoxynojirim...

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Detalles Bibliográficos
Autores principales:	Machaczka, Maciej, Hast, Robert, Dahlman, Ingrid, Lerner, Richard, Klimkowska, Monika, Engvall, Martin, Hägglund, Hans
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	Informa Healthcare 2012
Materias:	Original Articles
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3282239/ https://www.ncbi.nlm.nih.gov/pubmed/22247978 http://dx.doi.org/10.3109/03009734.2011.641609

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