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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Publicat a:Sci Rep
Autors principals: Lund, Troy C., Miller, Weston P., Liao, Ai Yin, Tolar, Jakub, Shanley, Ryan, Pasquali, Marzia, Sando, Nicole, Bigger, Brian W., Polgreen, Lynda E., Orchard, Paul J.
Format: Artigo
Idioma:Inglês
Publicat: Nature Publishing Group UK 2019
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Article
Accés en línia: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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