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Chapter-3.15 Pompe Disease

BOOK TITLE: PG Textbook of Pediatrics: General Pediatrics and Neonatology (Volume 1)

Author

1. Kishnani Priya S
2. Mori Mari

ISBN

9789351527251

DOI

10.5005/jp/books/12853_3.15

Edition

1/e

Publishing Year

2015

Pages

Author Affiliations

1. Duke University Medical Center, Durham, North Carolina, USA
2. Duke University Medical Center, Durham, North Carolina, USA

Chapter keywords

Pompe disease, neuromuscular disease, GAA deficiency, IOPD, LOPD, enzyme replacement therapy, ERT, alglucosidase alfa, muscle biopsy, respiratory muscle

Abstract

Abstract

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