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Article

Enzyme therapy and immune response in relation to CRIM status: the Dutch experience in classic infantile Pompe disease.

Journal of inherited metabolic disease - 1 Mar 2015

van Gelder Carin M, Hoogeveen-Westerveld Marianne, Kroos Marian A, Plug Iris, van der Ploeg Ans T, Reuser Arnold J J

Abstract excerpt

BACKGROUND: Enzyme-replacement therapy (ERT) in Pompe disease--an inherited metabolic disorder caused by acid α-glucosidase deficiency and characterized in infants by generalized muscle weakness and cardiomyopathy--can be complicated by immune responses. Infants that do not produce any endogenous acid α-glucosidase, so-called CRIM-negative patients, reportedly develop a strong response. We report the clinical...

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Identifiers

PMID: 24715333
DOI: 10.1007/s10545-014-9707-6
PMCID: PMC4341007

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PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 7 Sept 2026. Manifest publication-graph-cohort-2026-09-08.1.

Source receipt: pubmed, retrieved 7 Sept 2026.

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Enzyme therapy and immune response in relation to CRIM status: the Dutch experience in classic infantile Pompe disease.PMID 24715333Read ↗

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