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Subcutaneous administration of recombinant human acid α-glucosidase co-formulated with the pharmacological chaperone AT2220 leads to lysosomal uptake of rhGAA and glycogen reduction in disease-relevant tissues of mice with Pompe disease

✍ Scribed by Lun, Yi; Pellegrino, Lee; Xu, Su; Feng, Jessie; Soska, Rebecca; Frascella, Michelle; Garcia, Anadina; Flanagan, John J.; Lockhart, David J.; Valenzano, Kenneth J.; Khanna, Richie

Book ID: 122276221
Publisher: Elsevier Science
Year: 2014
Tongue: English
Weight: 48 KB
Volume: 111
Category: Article
ISSN: 1096-7192
DOI: 10.1016/j.ymgme.2013.12.167

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