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Eliglustat tartrate for the treatment of adults with type 1 Gaucher disease
The purpose of this article is to review eliglustat tartrate, a substrate reduction therapy, for the treatment of Gaucher disease type 1 (GD1). GD is an rare inborn error of metabolism caused by accumulation of lipid substrates such as glucosylceramide within the monocyte-macrophage system that affe...
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| 出版年: | Drug Des Devel Ther |
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| 主要な著者: | , |
| フォーマット: | Artigo |
| 言語: | Inglês |
| 出版事項: |
Dove Medical Press
2015
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| 主題: | |
| オンライン・アクセス: | https://ncbi.nlm.nih.gov/pmc/articles/PMC4554398/ https://ncbi.nlm.nih.gov/pubmed/26345314 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.2147/DDDT.S77760 |
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