Linked Life Data

18174963

Source:http://linkedlifedata.com/resource/pubmed/id/18174963

Statements in which the resource exists as a subject.
PredicateObject
rdf:type
lifeskim:mentions
pubmed:issue
11
pubmed:dateCreated
2008-1-4
pubmed:abstractText
Hunter syndrome (mucopolysaccharidosis II, MPS II) is a rare X-linked lysosomal storage disorder caused by the deficiency of enzyme iduronate-2-sulfatase (I2S), which results in accumulation of undegraded dermatan and heparan sulfate in various tissues and organs. Enzyme replacement therapy with Elaprase (idursulfase, a recently approved orphan product) is the first treatment for Hunter syndrome. Results of the randomized, double-blind, placebo-controlled phase II/III clinical trial of idursulfase demonstrated that weekly infusions of idursulfase increase walking distance and improve pulmonary function as well as reduce organ size and urinary glycosaminoglycans (GAGs) excretion in MPS II patients. Idursulfase is generally well tolerated, although infusion reactions do occur. Clinical studies demonstrate that idursulfase may be the first successful symptomatic therapy that can benefit patients with MPS II by addressing the enzymatic defect.
pubmed:language
eng
pubmed:journal
pubmed:citationSubset
IM
pubmed:chemical
pubmed:status
MEDLINE
pubmed:month
Nov
pubmed:issn
1699-3993
pubmed:author
pubmed:issnType
Print
pubmed:volume
43
pubmed:owner
NLM
pubmed:authorsComplete
Y
pubmed:pagination
759-67
pubmed:dateRevised
2008-7-25
pubmed:meshHeading
pubmed:year
2007
pubmed:articleTitle
Idursulfase in Hunter syndrome treatment.
pubmed:affiliation
Department of Environmental Medicine, University of Rochester, School of Medicine and Dentistry, Rochester, New York, USA. grazyna_zareba@urmc.rochester.edu
pubmed:publicationType
Journal Article, Review