Document Detail


Review of the clinical development of alipogene tiparvovec gene therapy for lipoprotein lipase deficiency.
MedLine Citation:
PMID:  20427244     Owner:  NLM     Status:  In-Data-Review    
Abstract/OtherAbstract:
Alipogene tiparvovec (AAV1-LPL(S447X)) gene therapy is developed to prevent complications and decrease the clinical morbidity of lipoprotein lipase deficiency (LPLD). LPLD is an autosomal recessive disease associated with severe hypertriglyceridemia (hyperTG), severe chylomicronaemia, and low HDL. Acute pancreatitis, the most frequent serious clinical LPLD complication, is a complex and heterogeneous inflammatory condition having many causes including hyperTG and chylomicronaemia. In many patients, low fat diet and currently available lipid lowering drugs are ineffective to prevent hyperTG or pancreatitis in LPLD. The clinical development program of alipogene tiparvovec includes observational studies as well as phase I/II and II/III clinical trials. Pooled data are collected on safety and efficacy issues, including the incidence of pancreatitis.
Authors:
Daniel Gaudet; Janneke de Wal; Karine Tremblay; Stéphane Déry; Sander van Deventer; Andreas Freidig; Diane Brisson; Julie Méthot
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Publication Detail:
Type:  Journal Article     Date:  2010-04-27
Journal Detail:
Title:  Atherosclerosis. Supplements     Volume:  11     ISSN:  1878-5050     ISO Abbreviation:  Atheroscler Suppl     Publication Date:  2010 Jun 
Date Detail:
Created Date:  2010-05-25     Completed Date:  -     Revised Date:  -    
Medline Journal Info:
Nlm Unique ID:  100973461     Medline TA:  Atheroscler Suppl     Country:  Netherlands    
Other Details:
Languages:  eng     Pagination:  55-60     Citation Subset:  IM    
Copyright Information:
Copyright © 2010 Elsevier Ireland Ltd. All rights reserved.
Affiliation:
Department of Medicine, Université de Montréal, ECOGENE-21 Clinical Research Center, Chicoutimi Hospital, Canada.
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