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The Immune Response to Intrathecal Enzyme Replacement Therapy in Mucopolysaccharidosis I Patients

BACKGROUND: Intrathecal (IT) enzyme replacement therapy with recombinant human α-l-iduronidase (rhIDU) has been studied to treat glycosaminoglycan storage in the central nervous system of mucopolysaccharidosis (MPS) I dogs and is currently being studied in MPS I patients. METHODS: We studied the imm...

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Autors principals: Vera, Moin, Le, Steven, Kan, Shih-hsin, Garban, Hermes, Naylor, David, Mlikotic, Anton, Kaitila, Ilkka, Harmatz, Paul, Chen, Agnes, Dickson, Patricia
Format: Artigo
Idioma:Inglês
Publicat: 2013
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Accés en línia:https://ncbi.nlm.nih.gov/pmc/articles/PMC3855632/
https://ncbi.nlm.nih.gov/pubmed/24002329
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/pr.2013.158
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