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Atypical immunologic response in a patient with CRIM-negative Pompe disease
We report the clinical course of a patient with severe infantile onset Pompe disease [cross-reactive immunologic material (CRIM) negative, R854X/R854X] who was diagnosed prenatally and received standard dosing of alglucosidase alfa (Myozyme®) enzyme replacement therapy (ERT) from day 10 of life unti...
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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/PMC3711241/ https://ncbi.nlm.nih.gov/pubmed/21889385 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.ymgme.2011.08.003 |
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