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Article

Cerebral magnetic resonance findings during enzyme replacement therapy in mucopolysaccharidosis.

Pediatric radiology - 1 Nov 2017

Matsubara Yoshiko, Miyazaki Osamu, Kosuga Motomichi, Okuyama Torayuki, Nosaka Shunsuke

Abstract excerpt

BACKGROUND: Although enzyme replacement therapy (ERT) is an effective treatment for mucopolysaccharidosis (MPS) types I, II, IVA and VI, its effectiveness in children with central nervous system (CNS) disorders is said to be poor because the blood-brain barrier cannot be penetrated by ERT drugs. OBJECTIVE: To assess CNS involvement in mucopolysaccharidosis at the start of enzyme replacement therapy and to...

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Identifiers

PMID: 28733853
DOI: 10.1007/s00247-017-3935-5

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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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