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Hurdles in treating Hurler disease: potential routes to achieve a “real” cure
Mucopolysaccharidoses (MPSs) are multiorgan devastating diseases for which hematopoietic cell transplantation (HCT) and, to a lesser extent, enzyme replacement therapy have substantially altered the course of the disease. Furthermore, they have resulted in increased overall survival, especially for...
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| 出版年: | Blood Adv |
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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 of Hematology
2020
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| 主題: | |
| オンライン･アクセス: | https://ncbi.nlm.nih.gov/pmc/articles/PMC7322951/ https://ncbi.nlm.nih.gov/pubmed/32574368 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1182/bloodadvances.2020001708 |
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