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Article

Enzyme replacement therapy in mucopolysaccharidosis type I.

Acta paediatrica (Oslo, Norway : 1992). Supplement - 1 Mar 2005

Miebach E

Abstract excerpt

UNLABELLED: Mucopolysaccharidosis (MPS) type I is a lysosomal storage disorder caused by deficiency of the enzyme alpha-L-iduronidase (IDUA), which presents with a wide spectrum of phenotypes. Recently, enzyme replacement therapy (ERT) became available for patients with MPS I and has been demonstrated to be safe and effective in patients with the milder Hurler-Scheie and Scheie phenotypes. Treatment for 26 weeks...

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Share to Bone Marrow Transplantation
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Source

Identifiers

PMID: 15895714
DOI: 10.1111/j.1651-2227.2005.tb02114.x

Genes

IDUA

Provenance

PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 7 Sept 2026. Manifest publication-graph-cohort-2026-09-07.1.

Source receipt: pubmed, retrieved 7 Sept 2026.

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