Linked Life Data

17546772

Source:http://linkedlifedata.com/resource/pubmed/id/17546772

Statements in which the resource exists as a subject.
PredicateObject
rdf:type
lifeskim:mentions
pubmed:dateCreated
2007-6-4
pubmed:abstractText
Preclinical and phase I/II studies gave the proof of principle of enzyme replacement therapy (ERT) with recombinant alpha-galactosidase A through the demonstration of the clearance of the accumulated subtrate from plasma and tissues. In a multicenter, randomized, placebo-controlled, double-blind phase Ill study, the biological efficacy of recombinant alpha-galactosidose A (agalsidase beta 1 mg/kg/714 days) was demonstrated on the basis of complete clearance of accumulated globotriaosylceramide from the endothelia of the kidney, heart and skin. The phase III extension study data gives additional results: kidney function appears to be stabilized after 54 to 60 months of treatment with agolsidase beta in most patients. Intent-to-treat analysis of a double-blind, randomized, placebo-controlled, phase IV study, showed that, adjusted for on imbalance in baseline proteinuria, agalsidase beta significantly reduces by 53% the risk of a first clinical event (renal, cardiac and cerebrovascular), compared with placebo. Clinical benefits of ERT depend on patients' clinical status at baseline, therefore prompting for onset of ERT before irreversible damage occur and underlying the need to stratify patients' populations to better understand the outcome of ERT.
pubmed:language
fre
pubmed:journal
pubmed:citationSubset
IM
pubmed:chemical
pubmed:status
MEDLINE
pubmed:month
Mar
pubmed:issn
0755-4982
pubmed:author
pubmed:issnType
Print
pubmed:volume
36 Spec No 1
pubmed:owner
NLM
pubmed:authorsComplete
Y
pubmed:pagination
1S69-75
pubmed:dateRevised
2007-11-15
pubmed:meshHeading
pubmed:year
2007
pubmed:articleTitle
[Development of an orphan drug to treat a genetic disease: the paradigm of agalsidase beta].
pubmed:affiliation
Centre de référence de la maladie de Fabry et des maladies héréditaires du tissu conjonctif (syndromes d'Ehlers-Danlos et pseudoxanthome élastique), Assistance Publique-Hôpitaux de Paris, Paris (75), France. dominique.germain@egp.aphp.fr
pubmed:publicationType
Journal Article, English Abstract, Review