Earlier and higher dosing of alglucosidase alfa improve outcomes in patients with infantile-onset Pompe disease: Evidence from real-world experiences

Published 2020 View Full Article

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Title
Earlier and higher dosing of alglucosidase alfa improve outcomes in patients with infantile-onset Pompe disease: Evidence from real-world experiences
Authors
Keywords
Newborn screening, Early treatment, Enzyme replacement therapy, Pompe disease, Dosage
Journal
Molecular Genetics and Metabolism Reports
Volume 23, Issue -, Pages 100591
Publisher
Elsevier BV
Online
2020-04-29
DOI
10.1016/j.ymgmr.2020.100591

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