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

Richie Khanna; John J. Flanagan; Jessie Feng; Rebecca Soska; Michelle Frascella; Lee J. Pellegrino; Yi Lun; Darlene Guillen; David J. Lockhart; Kenneth J. Valenzano

doi:10.1371/journal.pone.0040776

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Open Access

Peer-reviewed

Research Article

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

Richie Khanna ,

* E-mail: rkhanna@amicusrx.com

Affiliation Amicus Therapeutics Inc, Cranbury, New Jersey, United States of America
⨯
John J. Flanagan,

Affiliation Amicus Therapeutics Inc, Cranbury, New Jersey, United States of America
⨯
Jessie Feng,

Affiliation Amicus Therapeutics Inc, Cranbury, New Jersey, United States of America
⨯
Rebecca Soska,

Affiliation Amicus Therapeutics Inc, Cranbury, New Jersey, United States of America
⨯
Michelle Frascella,

Affiliation Amicus Therapeutics Inc, Cranbury, New Jersey, United States of America
⨯
Lee J. Pellegrino,

Affiliation Amicus Therapeutics Inc, Cranbury, New Jersey, United States of America
⨯
Yi Lun,

Affiliation Amicus Therapeutics Inc, Cranbury, New Jersey, United States of America
⨯
Darlene Guillen,

Affiliation Amicus Therapeutics Inc, Cranbury, New Jersey, United States of America
⨯
David J. Lockhart,

Affiliation Amicus Therapeutics Inc, Cranbury, New Jersey, United States of America
⨯
Kenneth J. Valenzano

Affiliation Amicus Therapeutics Inc, Cranbury, New Jersey, United States of America
⨯

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

Richie Khanna,
John J. Flanagan,
Jessie Feng,
Rebecca Soska,
Michelle Frascella,
Lee J. Pellegrino,
Yi Lun,
Darlene Guillen,
David J. Lockhart,
Kenneth J. Valenzano

Published: July 18, 2012
https://doi.org/10.1371/journal.pone.0040776

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Media Coverage of this Article

Posted by PLOS_ONE_Group on 04 Jan 2013 at 22:05 GMT

The following article represents some of the media coverage that has occurred for this paper:

Publication: Market Watch
Title: “Amicus Therapeutics Announces Positive Results From All Four Cohorts in Phase 2 Chaperone-Enzyme Replacement Therapy (ERT) Co-Administration Study for Pompe Disease - MarketWatch”
http://www.marketwatch.co...

If you see any additional coverage of this paper in the press or blogosphere, please reply to this thread and add the link to the article.

Competing interests declared: PLOS ONE Staff

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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

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

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Media Coverage of this Article

Posted by PLOS_ONE_Group on 04 Jan 2013 at 22:05 GMT