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The Pharmacological Chaperone AT2220 Increases the Specific Activity and Lysosomal Delivery of Mutant Acid Alpha-Glucosidase, and Promotes Glycogen Reduction in a Transgenic Mouse Model of Pompe Disease.
- Published in:
- PLoS ONE, 2014, v. 9, n. 7, p. 1, doi. 10.1371/journal.pone.0102092
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- Article
Migalastat HCl Reduces Globotriaosylsphingosine (Lyso-Gb<sub>3</sub>) in Fabry Transgenic Mice and in the Plasma of Fabry Patients.
- Published in:
- PLoS ONE, 2013, v. 8, n. 3, p. 1, doi. 10.1371/journal.pone.0057631
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- Article
The Pharmacological Chaperone AT2220 Increases Recombinant Human Acid α-Glucosidase Uptake and Glycogen Reduction in a Mouse Model of Pompe Disease.
- Published in:
- PLoS ONE, 2012, v. 7, n. 7, p. 1, doi. 10.1371/journal.pone.0040776
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- Article
Coformulation of a Novel Human α-Galactosidase A With the Pharmacological Chaperone AT1001 Leads to Improved Substrate Reduction in Fabry Mice.
- Published in:
- Molecular Therapy, 2015, v. 23, n. 7, p. 1169, doi. 10.1038/mt.2015.87
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- Article
The pharmacological chaperone isofagomine increases the activity of the Gaucher disease L444P mutant form of β-glucosidase.
- Published in:
- FEBS Journal, 2010, v. 277, n. 7, p. 1618, doi. 10.1111/j.1742-4658.2010.07588.x
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- Article
Co-administration With the Pharmacological Chaperone AT1001 Increases Recombinant Human α-Galactosidase A Tissue Uptake and Improves Substrate Reduction in Fabry Mice.
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- Molecular Therapy, 2012, v. 20, n. 4, p. 717, doi. 10.1038/mt.2011.271
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- Article
The Pharmacological Chaperone 1-Deoxygalactonojirimycin Reduces Tissue Globotriaosylceramide Levels in a Mouse Model of Fabry Disease.
- Published in:
- Molecular Therapy, 2010, v. 18, n. 1, p. 23, doi. 10.1038/mt.2009.220
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- Article