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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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| Main Authors: | , , , , , , |
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| Formato: | Artigo |
| Idioma: | Inglês |
| Publicado em: |
Informa Healthcare
2012
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| Assuntos: | |
| Acesso em linha: | https://ncbi.nlm.nih.gov/pmc/articles/PMC3282239/ https://ncbi.nlm.nih.gov/pubmed/22247978 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.3109/03009734.2011.641609 |
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