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Durable and sustained immune tolerance to ERT in Pompe disease with entrenched immune responses
BACKGROUND. Enzyme replacement therapy (ERT) has prolonged survival and improved clinical outcomes in patients with infantile Pompe disease (IPD), a rapidly progressive neuromuscular disorder. Yet marked interindividual variability in response to ERT, primarily attributable to the development of ant...
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| 出版年: | JCI Insight |
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| 主要な著者: | , , , , , , , , , |
| フォーマット: | Artigo |
| 言語: | Inglês |
| 出版事項: |
American Society for Clinical Investigation
2016
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| 主題: | |
| オンライン･アクセス: | https://ncbi.nlm.nih.gov/pmc/articles/PMC4970756/ https://ncbi.nlm.nih.gov/pubmed/27493997 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1172/jci.insight.86821 |
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