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Management Guidelines for Mucopolysaccharidosis VI.

Authors :
Giugliani, Roberto
Harmatz, Paul
Wraith, James E.
Source :
Pediatrics. Aug2007, Vol. 120 Issue 2, p405-418. 14p. 3 Black and White Photographs, 6 Diagrams, 2 Charts.
Publication Year :
2007

Abstract

Mucopolysaccharidosis VI (Maroteaux-Lamy syndrome) is a lysosomal storage disease that is characterized by systemic clinical manifestations and significant functional impairment. Diagnosis and management are often challenging because of the considerable variability in symptom presentation and rate of progression. The optimal standard of care should be based on evidence from randomized, controlled trials, meta-analyses, systematic reviews, and expert opinion. In support of this goal, comprehensive management guidelines have been drafted by an international group of experts in the management of patients with mucopolysaccharidosis VI. The guidelines provide a detailed outline of disease manifestations by body system, recommendations for regular assessments, and an overview of current treatment options. [ABSTRACT FROM AUTHOR]

Details

Language :
English
ISSN :
00314005
Volume :
120
Issue :
2
Database :
Academic Search Index
Journal :
Pediatrics
Publication Type :
Academic Journal
Accession number :
26364867
Full Text :
https://doi.org/10.1542/peds.2006-2184