| Review of the clinical development of alipogene tiparvovec gene therapy for lipoprotein lipase deficiency. | |
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MedLine Citation:
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PMID: 20427244 Owner: NLM Status: In-Data-Review |
Abstract/OtherAbstract:
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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. |
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Authors:
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Daniel Gaudet; Janneke de Wal; Karine Tremblay; Stéphane Déry; Sander van Deventer; Andreas Freidig; Diane Brisson; Julie Méthot |
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Publication Detail:
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Type: Journal Article Date: 2010-04-27 |
Journal Detail:
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Title: Atherosclerosis. Supplements Volume: 11 ISSN: 1878-5050 ISO Abbreviation: Atheroscler Suppl Publication Date: 2010 Jun |
Date Detail:
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Created Date: 2010-05-25 Completed Date: - Revised Date: - |
Medline Journal Info:
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Nlm Unique ID: 100973461 Medline TA: Atheroscler Suppl Country: Netherlands |
Other Details:
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Languages: eng Pagination: 55-60 Citation Subset: IM |
Copyright Information:
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Copyright © 2010 Elsevier Ireland Ltd. All rights reserved. |
Affiliation:
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Department of Medicine, Université de Montréal, ECOGENE-21 Clinical Research Center, Chicoutimi Hospital, Canada. |
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From MEDLINE®/PubMed®, a database of the U.S. National Library of Medicine
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