Found: 16
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Lysosomal Dysfunction in a Mouse Model of Sandhoff Disease Leads to Accumulation of Ganglioside-Bound Amyloid-ß Peptide.
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- Journal of Neuroscience, 2012, v. 32, n. 15, p. 5223, doi. 10.1523/JNEUROSCI.4860-11.2012
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- Article
Validation of Anti-Adeno Associated Virus Serotype rh10 (AAVrh.10) Total and Neutralizing Antibody Immunogenicity Assays.
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- Pharmaceutical Research, 2023, v. 40, n. 10, p. 2383, doi. 10.1007/s11095-023-03625-7
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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.
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- Molecular Therapy, 2010, v. 18, n. 1, p. 23, doi. 10.1038/mt.2009.220
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- Article
Migalastat Tissue Distribution: Extrapolation From Mice to Humans Using Pharmacokinetic Modeling and Comparison With Agalsidase Beta Tissue Distribution in Mice.
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- Clinical Pharmacology in Drug Development, 2021, v. 10, n. 9, p. 1075, doi. 10.1002/cpdd.941
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- Article
Pharmacological chaperones increase wild type presenilin 1 levels and promote the normalization of gamma-secretase function in presenilin 1 early-onset familial Alzheimer's disease models
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- 2012
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- Abstract
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.
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- PLoS ONE, 2014, v. 9, n. 7, p. 1, doi. 10.1371/journal.pone.0102092
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- Article
Immune Responses and Hypercoagulation in ERT for Pompe Disease Are Mutation and rhGAA Dose Dependent.
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- PLoS ONE, 2014, v. 9, n. 6, p. 1, doi. 10.1371/journal.pone.0098336
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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.
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- 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.
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- PLoS ONE, 2012, v. 7, n. 7, p. 1, doi. 10.1371/journal.pone.0040776
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- Article
Poly(A)-binding-protein-mediated regulation of hDcp2 decapping in vitro.
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- EMBO Journal, 2004, v. 23, n. 9, p. 1968, doi. 10.1038/sj.emboj.7600213
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- Article
Identification and Characterization of Pharmacological Chaperones to Correct Enzyme Deficiencies in Lysosomal Storage Disorders.
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- Assay & Drug Development Technologies, 2011, v. 9, n. 3, p. 213, doi. 10.1089/adt.2011.0370
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- Article
Expression and processing of mature human frataxin after gene therapy in mice.
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- Scientific Reports, 2024, v. 14, n. 1, p. 1, doi. 10.1038/s41598-024-59060-0
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- Article
Oral Migalastat HCl Leads to Greater Systemic Exposure and Tissue Levels of Active α-Galactosidase A in Fabry Patients when Co-Administered with Infused Agalsidase.
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- PLoS ONE, 2015, v. 10, n. 8, p. 1, doi. 10.1371/journal.pone.0134341
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- Article
Structure of acid β-glucosidase with pharmacological chaperone provides insight into Gaucher disease.
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- 2007
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- Publication type:
- Letter
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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- Publication type:
- Article