Abstract: P475 SAFETY AND EFFICACY OF AMT-010 GENE THERAPY IN LIPOPROTEIN LIPASE DEFICIENCY (LPLD)

E Stroes; J Kuivenhoven; S van Deventer; J Kastelein

doi:10.1016/s1567-5688(09)70770-6

Abstract: P475 SAFETY AND EFFICACY OF AMT-010 GENE THERAPY IN LIPOPROTEIN LIPASE DEFICIENCY (LPLD)

Atherosclerosis Supplements ◽

10.1016/s1567-5688(09)70770-6 ◽

2009 ◽

Vol 10 (2) ◽

pp. e786

Author(s):

E Stroes ◽

J Kuivenhoven ◽

S van Deventer ◽

J Kastelein

Keyword(s):

Gene Therapy ◽

Lipoprotein Lipase ◽

Safety And Efficacy ◽

Lipoprotein Lipase Deficiency

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Abstract: 554 AN OPEN-LABEL, DOSE ESCALATION STUDY TO ASSESS THE SAFETY AND EFFICACY OF AAV1-LPLS447X GENE THERAPY WITH ALIPOGENE TIPARVOVEC (AMT-011) FOR PATIENTS WITH SEVERE HYPERTRIGLYCERIDEMIA DUE TO LIPOPROTEIN LIPASE DEFICIENCY (LPLD)

Atherosclerosis Supplements ◽

10.1016/s1567-5688(09)70286-7 ◽

2009 ◽

Vol 10 (2) ◽

pp. e286 ◽

Cited By ~ 1

Author(s):

D Gaudet ◽

D Brisson ◽

J Methot ◽

S van Deventer

Keyword(s):

Gene Therapy ◽

Lipoprotein Lipase ◽

Dose Escalation ◽

Open Label ◽

Dose Escalation Study ◽

Safety And Efficacy ◽

Lipoprotein Lipase Deficiency ◽

Severe Hypertriglyceridemia ◽

Label Dose

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First global, longitudinal, pharmaco-epidemiologic, observational registry on gene therapy in the management of lipoprotein lipase deficiency (GENIALL)

Atherosclerosis ◽

10.1016/j.atherosclerosis.2015.04.482 ◽

2015 ◽

Vol 241 (1) ◽

pp. e139 ◽

Cited By ~ 1

Author(s):

E. Steinhagen-Thiessen ◽

E. Stroes ◽

H. Soran ◽

C. Johnson ◽

G. Iotti ◽

...

Keyword(s):

Gene Therapy ◽

Lipoprotein Lipase ◽

Lipoprotein Lipase Deficiency

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Long-Term Retrospective Analysis of Gene Therapy with Alipogene Tiparvovec and Its Effect on Lipoprotein Lipase Deficiency-Induced Pancreatitis

Human Gene Therapy ◽

10.1089/hum.2015.158 ◽

2016 ◽

Vol 27 (11) ◽

pp. 916-925 ◽

Cited By ~ 47

Author(s):

Daniel Gaudet ◽

Erik S. Stroes ◽

Julie Méthot ◽

Diane Brisson ◽

Karine Tremblay ◽

...

Keyword(s):

Gene Therapy ◽

Retrospective Analysis ◽

Lipoprotein Lipase ◽

Lipoprotein Lipase Deficiency

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Gene Therapy for Lipoprotein Lipase Deficiency: Working Toward Clinical Application

Human Gene Therapy ◽

10.1089/hum.2005.16.ft-138 ◽

2005 ◽

Vol 0 (0) ◽

pp. 051014061337001

Author(s):

Jaap Rip ◽

Melchior C. Nierman ◽

Jeroen A. Sierts ◽

Wilma Petersen ◽

Karin Van Den Oever ◽

...

Keyword(s):

Gene Therapy ◽

Lipoprotein Lipase ◽

Clinical Application ◽

Lipoprotein Lipase Deficiency

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Effect of gene therapy with alipogene tiparvovec on the incidence of pancreatitis in patients with lipoprotein lipase deficiency

Pancreatology ◽

10.1016/j.pan.2015.05.212 ◽

2015 ◽

Vol 15 (3) ◽

pp. S53-S54

Author(s):

Sophie J. Bernelot Moens ◽

Erik S. Stroes ◽

Marc Andersen ◽

Christian Meyer ◽

Diane Brisson ◽

...

Keyword(s):

Gene Therapy ◽

Lipoprotein Lipase ◽

Lipoprotein Lipase Deficiency

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Gene therapy for lipoprotein lipase deficiency

Current Opinion in Lipidology ◽

10.1097/mol.0b013e3283555a7e ◽

2012 ◽

Vol 23 (4) ◽

pp. 310-320 ◽

Cited By ~ 85

Author(s):

Daniel Gaudet ◽

Julie Méthot ◽

John Kastelein

Keyword(s):

Gene Therapy ◽

Lipoprotein Lipase ◽

Lipoprotein Lipase Deficiency

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Patient and Environmental Safety of AMT-010 Gene Therapy for Up To 4 Years in Lipoprotein Lipase Deficiency (LPLD)

Atherosclerosis Supplements ◽

10.1016/j.atherosclerosissup.2010.04.027 ◽

2010 ◽

Vol 11 (1) ◽

pp. 75

Author(s):

E.S. Stroes ◽

M. Sonnemans ◽

N. van den Bulk ◽

J.J.P. Kastelein

Keyword(s):

Gene Therapy ◽

Lipoprotein Lipase ◽

Environmental Safety ◽

Lipoprotein Lipase Deficiency

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Immune Responses to Intramuscular Administration of Alipogene Tiparvovec (AAV1-LPLS447X) in a Phase II Clinical Trial of Lipoprotein Lipase Deficiency Gene Therapy

Human Gene Therapy ◽

10.1089/hum.2013.169 ◽

2014 ◽

Vol 25 (3) ◽

pp. 180-188 ◽

Cited By ~ 79

Author(s):

Valerie Ferreira ◽

Jaap Twisk ◽

Karin Kwikkers ◽

Eleonora Aronica ◽

Diane Brisson ◽

...

Keyword(s):

Clinical Trial ◽

Gene Therapy ◽

Immune Responses ◽

Lipoprotein Lipase ◽

Phase Ii ◽

Phase Ii Clinical Trial ◽

Intramuscular Administration ◽

Lipoprotein Lipase Deficiency ◽

Deficiency Gene

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Gene therapy for lipoprotein lipase deficiency (LPLD): Final results of 3 prospective gene therapy clinical studies and 1 retrospective clinical events analysis

Atherosclerosis ◽

10.1016/j.atherosclerosis.2015.04.100 ◽

2015 ◽

Vol 241 (1) ◽

pp. e26

Author(s):

S.J. Bernelot-Moens ◽

E.S. Stroes ◽

A.C. Carpentier ◽

D. Brisson ◽

M. Andersen ◽

...

Keyword(s):

Gene Therapy ◽

Lipoprotein Lipase ◽

Clinical Studies ◽

Clinical Events ◽

Lipoprotein Lipase Deficiency

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Faculty Opinions recommendation of Review of the clinical development of alipogene tiparvovec gene therapy for lipoprotein lipase deficiency.

Faculty Opinions – Post-Publication Peer Review of the Biomedical Literature ◽

10.3410/f.12856959.14144057 ◽

2011 ◽

Author(s):

Margriet Vervoordeldonk ◽

Caroline Aalbers

Keyword(s):

Gene Therapy ◽

Lipoprotein Lipase ◽

Clinical Development ◽

Lipoprotein Lipase Deficiency

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