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Immune tolerance induction for laronidase treatment in mucopolysaccharidosis I

Enzyme replacement therapy (ERT) can produce anti-drug antibody (ADA) responses that reduce efficacy or lead to hypersensitivity reactions. Six patients with severe mucopolysaccharidosis type I (MPS I/Hurler syndrome) who did not receive hematopoietic stem cell transplantation underwent an immunosup...

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Podrobná bibliografie
Vydáno v:Mol Genet Metab Rep
Hlavní autoři: Giugliani, Roberto, Vieira, Taiane Alves, Carvalho, Clarissa Gutierrez, Muñoz-Rojas, Maria-Veronica, Semyachkina, Alla N., Voinova, Victoria Y., Richards, Susan, Cox, Gerald F., Xue, Yong
Médium: Artigo
Jazyk:Inglês
Vydáno: Elsevier 2017
Témata:
On-line přístup:https://ncbi.nlm.nih.gov/pmc/articles/PMC5238455/
https://ncbi.nlm.nih.gov/pubmed/28119821
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.ymgmr.2017.01.004
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