Strategy to assess the effect of duvoglustat co-administered with alglucosidase alfa infusion on the immune response to enzyme replacement therapy for Pompe disease

Elfrida R. Benjamin; Xiaoyang Wu; M. Cecilia Della Valle; Eric Sjoberg; Sheela Sitaraman; Anthony Stevens; Nestor Gomez; Evan Katz; Farhana Pruthi; Jeffry S. Kelley; Brandon Wustman; Kenneth J. Valenzano; Barry J. Byrne; Carrolee Barlow; David J. Lockhart

doi:10.1016/j.ymgme.2013.12.038

Strategy to assess the effect of duvoglustat co-administered with alglucosidase alfa infusion on the immune response to enzyme replacement therapy for Pompe disease

Molecular Genetics and Metabolism ◽

10.1016/j.ymgme.2013.12.038 ◽

2014 ◽

Vol 111 (2) ◽

pp. S25

Author(s):

Elfrida R. Benjamin ◽

Xiaoyang Wu ◽

M. Cecilia Della Valle ◽

Eric Sjoberg ◽

Sheela Sitaraman ◽

...

Keyword(s):

Immune Response ◽

Enzyme Replacement Therapy ◽

Replacement Therapy ◽

Pompe Disease ◽

Enzyme Replacement ◽

Alglucosidase Alfa

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A 4-year follow-up study of 24 patients with late onset Pompe disease treated with alglucosidase alfa enzyme replacement therapy at a single centre

Molecular Genetics and Metabolism ◽

10.1016/j.ymgme.2014.12.252 ◽

2015 ◽

Vol 114 (2) ◽

pp. S111

Author(s):

Karolina M. Stepien ◽

Jane Whitby ◽

Mark Roberts ◽

Reena Sharma

Keyword(s):

Enzyme Replacement Therapy ◽

Replacement Therapy ◽

Pompe Disease ◽

Late Onset ◽

Single Centre ◽

Enzyme Replacement ◽

Follow Up Study ◽

Alglucosidase Alfa

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Proteomics to identify signature proteins in patients likely to mount an immune response to enzyme replacement therapy in infantile Pompe disease

Molecular Genetics and Metabolism ◽

10.1016/j.ymgme.2014.12.128 ◽

2015 ◽

Vol 114 (2) ◽

pp. S61

Author(s):

Zoheb Kazi ◽

Katie Berrier ◽

Cheng Lu ◽

Deeksha Bali ◽

J. Will Thompson ◽

...

Keyword(s):

Immune Response ◽

Enzyme Replacement Therapy ◽

Replacement Therapy ◽

Pompe Disease ◽

Enzyme Replacement ◽

Infantile Pompe Disease

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Enzyme replacement therapy with alglucosidase alfa in Pompe disease: Clinical experience with rate escalation

Molecular Genetics and Metabolism ◽

10.1016/j.ymgme.2017.12.435 ◽

2018 ◽

Vol 123 (2) ◽

pp. 92-96 ◽

Cited By ~ 3

Author(s):

Ankit K. Desai ◽

Crista K. Walters ◽

Heidi L. Cope ◽

Zoheb B. Kazi ◽

Stephanie M. DeArmey ◽

...

Keyword(s):

Enzyme Replacement Therapy ◽

Replacement Therapy ◽

Clinical Experience ◽

Pompe Disease ◽

Enzyme Replacement ◽

Alglucosidase Alfa

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Characterization of immune response in Cross-Reactive Immunological Material (CRIM)-positive infantile Pompe disease patients treated with enzyme replacement therapy

Molecular Genetics and Metabolism Reports ◽

10.1016/j.ymgmr.2019.100475 ◽

2019 ◽

Vol 20 ◽

pp. 100475 ◽

Cited By ~ 3

Author(s):

Ankit K. Desai ◽

Zoheb B. Kazi ◽

Deeksha S. Bali ◽

Priya S. Kishnani

Keyword(s):

Immune Response ◽

Enzyme Replacement Therapy ◽

Replacement Therapy ◽

Pompe Disease ◽

Enzyme Replacement ◽

Infantile Pompe Disease

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Long‐term benefit of enzyme replacement therapy with alglucosidase alfa in adults with Pompe disease: Prospective analysis from the French Pompe Registry

Journal of Inherited Metabolic Disease ◽

10.1002/jimd.12272 ◽

2020 ◽

Vol 43 (6) ◽

pp. 1219-1231 ◽

Cited By ~ 1

Author(s):

Claudio Semplicini ◽

Marie De Antonio ◽

Nadjib Taouagh ◽

Anthony Béhin ◽

Françoise Bouhour ◽

...

Keyword(s):

Enzyme Replacement Therapy ◽

Replacement Therapy ◽

Pompe Disease ◽

Prospective Analysis ◽

Enzyme Replacement ◽

Alglucosidase Alfa ◽

Term Benefit

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B-Cell Depletion and Immunomodulation before Initiation of Enzyme Replacement Therapy Blocks the Immune Response to Acid Alpha-Glucosidase in Infantile-Onset Pompe Disease

The Journal of Pediatrics ◽

10.1016/j.jpeds.2013.03.002 ◽

2013 ◽

Vol 163 (3) ◽

pp. 847-854.e1 ◽

Cited By ~ 48

Author(s):

Melissa E. Elder ◽

Sushrusha Nayak ◽

Shelley W. Collins ◽

Lee Ann Lawson ◽

Jeffry S. Kelley ◽

...

Keyword(s):

Immune Response ◽

Enzyme Replacement Therapy ◽

B Cell ◽

Replacement Therapy ◽

Pompe Disease ◽

Cell Depletion ◽

B Cell Depletion ◽

Enzyme Replacement ◽

Alpha Glucosidase

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Phase 1 safety and pharmacokinetics of the novel enzyme replacement therapy neoGAA in treatment-naïve and alglucosidase alfa-treated late-onset Pompe disease patients

Molecular Genetics and Metabolism ◽

10.1016/j.ymgme.2015.12.476 ◽

2016 ◽

Vol 117 (2) ◽

pp. S117-S118

Author(s):

Shafeeq Ladha ◽

Pascal Laforet ◽

Eugen Mengel ◽

Alan Pestronk ◽

Jean Pouget ◽

...

Keyword(s):

Enzyme Replacement Therapy ◽

Replacement Therapy ◽

Pompe Disease ◽

Late Onset ◽

Phase 1 ◽

The Novel ◽

Enzyme Replacement ◽

Alglucosidase Alfa ◽

Treatment Naïve

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Unwarranted, long term, alglucosidase alfa enzyme replacement therapy in two non-Pompe disease patients

Clinical Neurology and Neurosurgery ◽

10.1016/j.clineuro.2020.106048 ◽

2020 ◽

Vol 196 ◽

pp. 106048

Author(s):

Chafic Karam ◽

Thomas Ragole ◽

Orly Moshe-Lilie ◽

Nizar Chahin

Keyword(s):

Enzyme Replacement Therapy ◽

Replacement Therapy ◽

Pompe Disease ◽

Enzyme Replacement ◽

Alglucosidase Alfa

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Phase 1 exploratory efficacy of the novel enzyme replacement therapy neoGAA in treatment-naïve and alglucosidase alfa-treated late-onset Pompe disease patients

Molecular Genetics and Metabolism ◽

10.1016/j.ymgme.2015.12.396 ◽

2016 ◽

Vol 117 (2) ◽

pp. S92

Author(s):

Loren Pena ◽

Richard Baron ◽

Barry Byrne ◽

Claude Desnuelle ◽

Ozlem Goker-Alpan ◽

...

Keyword(s):

Enzyme Replacement Therapy ◽

Replacement Therapy ◽

Pompe Disease ◽

Late Onset ◽

Phase 1 ◽

The Novel ◽

Enzyme Replacement ◽

Alglucosidase Alfa ◽

Treatment Naïve

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Cost-effectiveness of enzyme replacement therapy with alglucosidase alfa in classic-infantile patients with Pompe disease

Orphanet Journal of Rare Diseases ◽

10.1186/1750-1172-9-75 ◽

2014 ◽

Vol 9 (1) ◽

pp. 75 ◽

Cited By ~ 20

Author(s):

Tim A Kanters ◽

Iris Hoogenboom-Plug ◽

Maureen PMH Rutten-Van Mölken ◽

W Redekop ◽

Ans T van der Ploeg ◽

...

Keyword(s):

Cost Effectiveness ◽

Enzyme Replacement Therapy ◽

Replacement Therapy ◽

Pompe Disease ◽

Enzyme Replacement ◽

Alglucosidase Alfa

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