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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
- Source :
- Molecular Genetics and Metabolism. 111:S73
- Publication Year :
- 2014
- Publisher :
- Elsevier BV, 2014.
Details
- ISSN :
- 10967192
- Volume :
- 111
- Database :
- OpenAIRE
- Journal :
- Molecular Genetics and Metabolism
- Accession number :
- edsair.doi...........bcfbb28a7d24f860c64576b86b1561d2
- Full Text :
- https://doi.org/10.1016/j.ymgme.2013.12.167