Linked Life Data

22013559

Source:http://linkedlifedata.com/resource/pubmed/id/22013559

Statements in which the resource exists as a subject.
PredicateObject
rdf:type
lifeskim:mentions
pubmed:issue
7
pubmed:dateCreated
2011-10-20
pubmed:abstractText
Type 1 Gaucher disease (GD) results from inherited ?-glucocerebrosidase gene mutations, leading to anemia, thrombocytopenia, splenomegaly, hepatomegaly and skeletal disease. Velaglucerase alfa is a ?-glucocerebrosidase produced by gene activation in a human cell line, and indicated for type 1 GD. A phase I/II clinical trial (TKT025; N = 12), its ongoing extension (TKT025EXT) and three phase III trials (total N = 82), showed that velaglucerase alfa is generally well tolerated in adult and pediatric patients. Many disease-related parameters improved significantly in two phase III trials in treatment-naïve patients, and were successfully maintained in imiglucerase-experienced patients in a phase II/III switch study. Ten adults in TKT025EXT sustained improvements through 5 years, including bone mineral density. Comparison with imiglucerase shows that velaglucerase alfa is an effective, generally well-tolerated alternative enzyme replacement therapy. In vitro data suggest velaglucerase alfa may be internalized into cells more efficiently and have a lower rate of seroconversion. However, these results do not necessarily correlate with clinical efficacy.
pubmed:language
eng
pubmed:journal
pubmed:citationSubset
IM
pubmed:chemical
pubmed:status
MEDLINE
pubmed:month
Jul
pubmed:issn
1699-3993
pubmed:author
pubmed:copyrightInfo
Copyright 2011 Prous Science, S.A.U. or its licensors. All rights reserved.
pubmed:issnType
Print
pubmed:volume
47
pubmed:owner
NLM
pubmed:authorsComplete
Y
pubmed:pagination
515-29
pubmed:meshHeading
pubmed:year
2011
pubmed:articleTitle
Velaglucerase alfa: a new option for Gaucher disease treatment.
pubmed:affiliation
Shaare Zedek Medical Center, Jerusalem, Israel. azimran@gmail.com
pubmed:publicationType
Journal Article, Research Support, Non-U.S. Gov't