Phosphatidylserine Converts Immunogenic Recombinant Human Acid Alpha-Glucosidase to a Tolerogenic Form in a Mouse Model of Pompe Disease

Journal of Pharmaceutical Sciences ◽

10.1016/j.xphs.2016.06.018 ◽

2016 ◽

Vol 105 (10) ◽

pp. 3097-3104 ◽

Author(s):

Jennifer L. Schneider ◽

Sathy V. Balu-Iyer

Keyword(s):

Mouse Model ◽

Pompe Disease ◽

Alpha Glucosidase

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Stabilized next-generation recombinant human acid alpha-glucosidase ATB200 clears accumulated glycogen and reverses cellular dysfunction to increase functional muscle strength in a mouse model of Pompe disease

Molecular Genetics and Metabolism ◽

10.1016/j.ymgme.2016.11.083 ◽

2017 ◽

Vol 120 (1-2) ◽

pp. S42

Author(s):

Hung Do ◽

Yi Lun ◽

Su Xu ◽

Rebecca Soska ◽

Anju Nair ◽

...

Keyword(s):

Muscle Strength ◽

Mouse Model ◽

Pompe Disease ◽

Next Generation ◽

Cellular Dysfunction ◽

Alpha Glucosidase

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Stabilized next-generation recombinant human acid alpha-glucosidase ATB200 clears accumulated glycogen and reverses cellular dysfunction to increase muscle strength in a mouse model of Pompe disease

Neuromuscular Disorders ◽

10.1016/j.nmd.2017.06.247 ◽

2017 ◽

Vol 27 ◽

pp. S161

Author(s):

S. Xu ◽

Y. Lun ◽

A. Nair ◽

M. Frascella ◽

A. Garcia ◽

...

Keyword(s):

Muscle Strength ◽

Mouse Model ◽

Pompe Disease ◽

Next Generation ◽

Cellular Dysfunction ◽

Alpha Glucosidase

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P.17.8 The co-formulation of pharmacological chaperone AT2220 with recombinant human acid alpha-glucosidase improves enzyme uptake and glycogen reduction in a mouse model of Pompe disease

Neuromuscular Disorders ◽

10.1016/j.nmd.2013.06.658 ◽

2013 ◽

Vol 23 (9-10) ◽

pp. 828-829

Author(s):

R. Khanna ◽

S. Xu ◽

L. Pellegrino ◽

Y. Lun ◽

R. Soska ◽

...

Keyword(s):

Mouse Model ◽

Pompe Disease ◽

Pharmacological Chaperone ◽

Alpha Glucosidase

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Preliminary clinical efficacy and safety of BMN 701, GILT-tagged recombinant human acid alpha glucosidase (rhGAA), in late-onset Pompe disease: results of an extension study

Molecular Genetics and Metabolism ◽

10.1016/j.ymgme.2013.12.050 ◽

2014 ◽

Vol 111 (2) ◽

pp. S29

Author(s):

Barry Byrne ◽

Richard Barohn ◽

Bruce Barshop ◽

Drago Bratkovic ◽

Claude Desnuelle ◽

...

Keyword(s):

Clinical Efficacy ◽

Pompe Disease ◽

Extension Study ◽

Efficacy And Safety ◽

Alpha Glucosidase

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Four unreported types of glycans containing mannose-6-phosphate are heterogeneously attached at three sites (including newly found Asn 233) to recombinant human acid alpha-glucosidase that is the only approved treatment for Pompe disease

Biochemical and Biophysical Research Communications ◽

10.1016/j.bbrc.2017.12.101 ◽

2018 ◽

Vol 495 (4) ◽

pp. 2418-2424 ◽

Author(s):

Heajin Park ◽

Jihye Kim ◽

Young Kwang Lee ◽

Wooseok Kim ◽

Seung Kwan You ◽

...

Keyword(s):

Pompe Disease ◽

Mannose 6 Phosphate ◽

Alpha Glucosidase

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Pregnancy in a Case of Pompe Disease Treated with Recombinant Human Acid Alpha Glucosidase (Myozyme)

Clinical Therapeutics ◽

10.1016/j.clinthera.2011.05.063 ◽

2011 ◽

Vol 33 (6) ◽

pp. S22

Author(s):

F. Zagnoli ◽

A. Leblanc ◽

C. Blanchard ◽

A. Philippe ◽

M. Lastennet

Keyword(s):

Pompe Disease ◽

Alpha Glucosidase

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Benefit of Recombinant Human Acid Alpha Glucosidase Treatment (Myozyme) in Late Onset Pompe Disease: About Five Cases Treated for 3 Years

Clinical Therapeutics ◽

10.1016/j.clinthera.2011.05.064 ◽

2011 ◽

Vol 33 (6) ◽

pp. S23

Author(s):

F. Zagnoli ◽

A. Leblanc ◽

C. Blanchard ◽

A. Philippe ◽

M. Lastennet

Keyword(s):

Pompe Disease ◽

Alpha Glucosidase

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P287 – 1966 Pompe disease (PD): clinical outcome in infants and children treated with recombinant human acid alpha-glucosidase (rhGAA)

European Journal of Paediatric Neurology ◽

10.1016/s1090-3798(13)70466-5 ◽

2013 ◽

Vol 17 ◽

pp. S132

Author(s):

D Lianou ◽

D Syrengelas ◽

N Andreou ◽

I Chantzopoulos ◽

I Mavridou ◽

...

Keyword(s):

Clinical Outcome ◽

Pompe Disease ◽

Infants And Children ◽

Alpha Glucosidase ◽

In Infants And Children

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Report of the first Brazilian infantile Pompe disease patient to be treated with recombinant human acid alpha-glucosidase

Jornal de Pediatria ◽

10.2223/jped.1793 ◽

2008 ◽

Vol 84 (3) ◽

pp. 272-275 ◽

Author(s):

Sandra J. Pereira ◽

Célia R. Berditchevisky

Keyword(s):

Pompe Disease ◽

Disease Patient ◽

Infantile Pompe Disease ◽

Alpha Glucosidase

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The Pharmacological Chaperone AT2220 Increases Recombinant Human Acid α-Glucosidase Uptake and Glycogen Reduction in a Mouse Model of Pompe Disease

PLoS ONE ◽

10.1371/journal.pone.0040776 ◽

2012 ◽

Vol 7 (7) ◽

pp. e40776 ◽

Author(s):

Richie Khanna ◽

John J. Flanagan ◽

Jessie Feng ◽

Rebecca Soska ◽

Michelle Frascella ◽

...

Keyword(s):

Mouse Model ◽

Pompe Disease ◽

Pharmacological Chaperone ◽

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