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Article

Does early use of enzyme replacement therapy alter the natural history of mucopolysaccharidosis I? Experience in three siblings.

Molecular genetics and metabolism - 1 Jul 2013

Laraway Sarah, Breen Catherine, Mercer Jean, Jones Simon, Wraith James E

Abstract excerpt

Enzyme replacement therapy is widely used as treatment for mucopolysaccharidosis I (MPS I), and there is evidence that this produces improvement in certain clinical domains. There does appear to be variation in the response of clinical features to treatment once these are established. In a report...

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Identifiers

PMID: 23721889
DOI: 10.1016/j.ymgme.2013.04.023

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PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 4 Sept 2026. Prepared record publication-23721889-2026-09-04.2. Manifest publication-graph-cohort-2026-09-04.2.

Source receipt: PubMed via Helena Literature Corpus, retrieved 4 Sept 2026.

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