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Post-transplant laronidase augmentation for children with Hurler syndrome: biochemical outcomes
Allogeneic hematopoietic cell transplantation (HCT) benefits children with Hurler syndrome (MPS-IH). However, survivors remain burdened by substantial MPS-IH related residual disease. We studied the feasibility, safety and biochemical impact of augmentative recombinant intravenous enzyme replacement...
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| Publicado no: | Sci Rep |
|---|---|
| Main Authors: | , , , , , , , , , |
| Formato: | Artigo |
| Idioma: | Inglês |
| Publicado em: |
Nature Publishing Group UK
2019
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| Assuntos: | |
| Acesso em linha: | https://ncbi.nlm.nih.gov/pmc/articles/PMC6773848/ https://ncbi.nlm.nih.gov/pubmed/31575939 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/s41598-019-50595-1 |
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