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Immune Response to IT ERT in Mucopolysaccharidosis I Patient

ABSTRACT:

In this study we characterized the immune response to intrathecal (IT) recombinant human alpha-L-iduronidase (rhIDU) in mucopolysaccharidosis I (MPS I) subjects with spinal cord compression who had been previously treated with intravenous rhIDU. Concentrations of specific antibodies and cytokines were measured in serum and cerebrospinal fluid (CSF) collected before monthly IT rhIDU infusions. These serologic findings were compared with clinical adverse event (AE) reports to establish temporal correlations with clinical symptoms. We found that IT rhIDU was generally well tolerated in the subjects studied although one subject had moderate to severe clinical symptoms and serologic abnormalities consistent with an immune response.

PROVIDER: phs000862 | dbGaP |

SECONDARY ACCESSION(S): PRJNA189557PRJNA189556

REPOSITORIES: dbGaP

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			Action	DRS
	GapExchange_phs000862.v1.p1.xml	Xml
	dbGaPEx2.1.5.xsd	Other
	phs000862.v1-Documents.zip	Other
	Study_Report.phs000862.LDN_6719.v1.p1.MULTI.pdf	Pdf
	manifest_phs000862.LDN_6719.v1.p1.c1.GRU-IRB-PUB.pdf	Pdf

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