Mucopolysaccharidosis I: management and treatment guidelines.

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Abstract

Disease management for mucopolysaccharidosis type I has been inconsistent because of disease rarity (approximately 1 case per 100,000 live births), phenotypic heterogeneity, and limited therapeutic options. The availability of hematopoietic stem cell transplantation and the recent introduction of enzyme replacement therapy for mucopolysaccharidosis I necessitate the establishment of system-specific management guidelines for this condition.

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Journal

Journal ID (iso-abbrev): Pediatrics

Title: Pediatrics

Publisher: American Academy of Pediatrics (AAP)

ISSN (Electronic): 1098-4275

ISSN (Print): 0031-4005

Publication date (Electronic): Jan 2009

Volume: 123

Issue: 1

Affiliations

[1 ] Department of Pediatrics, University of North Carolina at Chapel Hill, Chapel Hill, NC 27514, USA. muenzer@med.unc.edu

Article

Publisher Item ID: 123/1/19

DOI: 10.1542/peds.2008-0416

PubMed ID: 19117856

SO-VID: 709ca963-dd5f-4ecb-a07d-539619bd992d

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