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Immune responses and hypercoagulation in ERT for Pompe disease are mutation and rhGAA dose dependent

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Record title

Immune responses and hypercoagulation in ERT for Pompe disease are mutation and rhGAA dose dependent

Record identifier

TN_cdi_plos_journals_1532654506

Immune responses and hypercoagulation in ERT for Pompe disease are mutation and rhGAA dose dependent

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https://collection.sl.nsw.gov.au/record/TN_cdi_plos_journals_1532654506

Immune responses and hypercoagulation in ERT for Pompe disease are mutation and rhGAA dose dependent

Full title

Immune responses and hypercoagulation in ERT for Pompe disease are mutation and rhGAA dose dependent

Publisher

United States: Public Library of Science

Journal title

PloS one, 2014, Vol.9 (6), p.e98336

Record Identifier

TN_cdi_plos_journals_1532654506

Language

English

Formats

Publication information

Publisher

United States: Public Library of Science

More information

SCOPE AND CONTENTS

Contents

Enzyme replacement therapy (ERT) with recombinant human acid-α-glucosidase (rhGAA) is the only FDA approved therapy for Pompe disease. Without ERT, severely affected individuals (early onset) succumb to the disease within 2 years of life. A spectrum of disease severity and progression exists depending upon the type of mutation in the GAA gene (GAA)...

ALTERNATIVE TITLES

Full title

Immune responses and hypercoagulation in ERT for Pompe disease are mutation and rhGAA dose dependent

Identifiers

PRIMARY IDENTIFIERS

Record Identifier

TN_cdi_plos_journals_1532654506

Permalink

https://collection.sl.nsw.gov.au/record/TN_cdi_plos_journals_1532654506

OTHER IDENTIFIERS

ISSN

1932-6203

E-ISSN

1932-6203

DOI

10.1371/journal.pone.0098336

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