The pharmacological chaperone AT2220 increases recombinant human acid α-glucosidase uptake and glycogen reduction in a mouse model of Pompe disease.
about
Targeted approaches to induce immune tolerance for Pompe disease therapyA lysosome-centered view of nutrient homeostasisThe 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 diseaseImmune responses and hypercoagulation in ERT for Pompe disease are mutation and rhGAA dose dependent.Antisense Oligonucleotide-mediated Suppression of Muscle Glycogen Synthase 1 Synthesis as an Approach for Substrate Reduction Therapy of Pompe DiseaseThe identification of the endogenous ligands of natural killer T cells reveals the presence of mammalian α-linked glycosylceramides.Enzyme replacement therapy and beyond-in memoriam Roscoe O. Brady, M.D. (1923-2016).Candidate molecules for chemical chaperone therapy of GM1-gangliosidosis.Enhanced efficacy from gene therapy in Pompe disease using coreceptor blockadePompe disease: literature review and case seriesPharmacological chaperone therapy for lysosomal storage diseases.A chaperone enhances blood α-glucosidase activity in Pompe disease patients treated with enzyme replacement therapy.Comparative impact of AAV and enzyme replacement therapy on respiratory and cardiac function in adult Pompe miceCoformulation of a Novel Human α-Galactosidase A With the Pharmacological Chaperone AT1001 Leads to Improved Substrate Reduction in Fabry MiceEffects of a higher dose of alglucosidase alfa on ventilator-free survival and motor outcome in classic infantile Pompe disease: an open-label single-center studyA thermodynamic assay to test pharmacological chaperones for Fabry disease.Glucocerebrosidase inhibitors for the treatment of Gaucher disease.Innovative strategies to treat protein misfolding in inborn errors of metabolism: pharmacological chaperones and proteostasis regulators.Natural product and natural product derived drugs in clinical trials.Myocardial glycogen dynamics: new perspectives on disease mechanisms.Pharmacological Chaperone Therapy: Preclinical Development, Clinical Translation, and Prospects for the Treatment of Lysosomal Storage Disorders.Glycomimetic-based pharmacological chaperones for lysosomal storage disorders: lessons from Gaucher, GM1-gangliosidosis and Fabry diseases.Pompe Disease: Diagnosis and Management. Evidence-Based Guidelines from a Canadian Expert Panel.Duvoglustat HCl Increases Systemic and Tissue Exposure of Active Acid α-Glucosidase in Pompe Patients Co-administered with Alglucosidase α.A Neuron-Specific Gene Therapy Relieves Motor Deficits in Pompe Disease Mice.Enzyme enhancers for the treatment of Fabry and Pompe disease.Pharmacoperones as Novel Therapeutics for Diverse Protein Conformational Diseases.
P2860
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P2860
The pharmacological chaperone AT2220 increases recombinant human acid α-glucosidase uptake and glycogen reduction in a mouse model of Pompe disease.
description
2012 nî lūn-bûn
@nan
2012 թուականի Յուլիսին հրատարակուած գիտական յօդուած
@hyw
2012 թվականի հուլիսին հրատարակված գիտական հոդված
@hy
2012年の論文
@ja
2012年論文
@yue
2012年論文
@zh-hant
2012年論文
@zh-hk
2012年論文
@zh-mo
2012年論文
@zh-tw
2012年论文
@wuu
name
The pharmacological chaperone ...... mouse model of Pompe disease.
@ast
The pharmacological chaperone ...... mouse model of Pompe disease.
@en
The pharmacological chaperone ...... mouse model of Pompe disease.
@nl
type
label
The pharmacological chaperone ...... mouse model of Pompe disease.
@ast
The pharmacological chaperone ...... mouse model of Pompe disease.
@en
The pharmacological chaperone ...... mouse model of Pompe disease.
@nl
prefLabel
The pharmacological chaperone ...... mouse model of Pompe disease.
@ast
The pharmacological chaperone ...... mouse model of Pompe disease.
@en
The pharmacological chaperone ...... mouse model of Pompe disease.
@nl
P2093
P2860
P1433
P1476
The pharmacological chaperone ...... mouse model of Pompe disease.
@en
P2093
Darlene Guillen
David J Lockhart
Jessie Feng
John J Flanagan
Kenneth J Valenzano
Lee J Pellegrino
Michelle Frascella
Rebecca Soska
Richie Khanna
P2860
P304
P356
10.1371/JOURNAL.PONE.0040776
P407
P577
2012-07-18T00:00:00Z