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Consensus Conference: A reappraisal of Gaucher disease - diagnosis and disease management algorithms
Type 1 (non neuronopathic) Gaucher disease was the first lysosomal storage disorder for which an effective enzyme replacement therapy was developed and it has become a prototype for treatments for related orphan diseases. There are currently four treatment options available to patients with Gaucher...
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| 主要な著者: | , , , , , , , , , , |
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| フォーマット: | Artigo |
| 言語: | Inglês |
| 出版事項: |
2011
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| 主題: | |
| オンライン・アクセス: | https://ncbi.nlm.nih.gov/pmc/articles/PMC3058841/ https://ncbi.nlm.nih.gov/pubmed/21080341 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1002/ajh.21888 |
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