Observational clinical study of 22 adult-onset Pompe disease patients undergoing enzyme replacement therapy over 5years

Published 2016 View Full Article

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Title
Observational clinical study of 22 adult-onset Pompe disease patients undergoing enzyme replacement therapy over 5years
Authors
Keywords
Late-onset Pompe disease, LOPD, Enzyme replacement therapy, Alpha glucosidase deficiency, Myozyme
Journal
MOLECULAR GENETICS AND METABOLISM
Volume 117, Issue 4, Pages 413-418
Publisher
Elsevier BV
Online
2016-02-05
DOI
10.1016/j.ymgme.2016.01.013

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