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Article

Effects of idursulfase enzyme replacement therapy for Mucopolysaccharidosis type II when started in early infancy: comparison in two siblings.

Molecular genetics and metabolism - 1 Mar 2013

Tajima Go, Sakura Nobuo, Kosuga Motomichi, Okuyama Torayuki, Kobayashi Masao

Abstract excerpt

Mucopolysaccharidosis type II (MPS II) is a lysosomal storage disorder that is progressive and involves multiple organs and tissues. While enzyme replacement therapy (ERT) with idursulfase has been shown to improve many somatic features of the disease, some such as dysostosis multiplex and cardiac valve disease appear irreversible once established, and little is known about the preventative effects of ERT in...

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PMID: 23375472
DOI: 10.1016/j.ymgme.2012.12.010

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IDS

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PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 5 Sept 2026. Manifest publication-graph-cohort-2026-09-05.1.

Source receipt: pubmed, retrieved 5 Sept 2026.

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