Pharmacological chaperone therapy for Gaucher disease: a patent review.
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Miglustat Reverts the Impairment of Synaptic Plasticity in a Mouse Model of NPC DiseaseCyclodextrin-mediated crystallization of acid β-glucosidase in complex with amphiphilic bicyclic nojirimycin analoguesPharmacological chaperones for human -N-acetylgalactosaminidaseGlycosidase inhibitors: a patent review (2008-2013).Rapid assembly of a library of lipophilic iminosugars via the thiol-ene reaction yields promising pharmacological chaperones for the treatment of Gaucher diseaseDiscovery, structure-activity relationship, and biological evaluation of noninhibitory small molecule chaperones of glucocerebrosidaseNeuronopathic Gaucher's disease: induced pluripotent stem cells for disease modelling and testing chaperone activity of small compounds.The multivalent effect in glycosidase inhibition: probing the influence of valency, peripheral ligand structure, and topology with cyclodextrin-based iminosugar click clusters.Pharmacological Chaperones and Coenzyme Q10 Treatment Improves Mutant β-Glucocerebrosidase Activity and Mitochondrial Function in Neuronopathic Forms of Gaucher Disease.Stereodivergent synthesis of right- and left-handed iminoxylitol heterodimers and monomers. Study of their impact on β-glucocerebrosidase activity.Characterization of the complex formed by β-glucocerebrosidase and the lysosomal integral membrane protein type-2.CNS-accessible Inhibitor of Glucosylceramide Synthase for Substrate Reduction Therapy of Neuronopathic Gaucher Disease.A thermodynamic assay to test pharmacological chaperones for Fabry disease.Gaucher disease paradigm: from ERAD to comorbidity.Cyclodextrin-based multivalent glycodisplays: covalent and supramolecular conjugates to assess carbohydrate-protein interactions.Glucocerebrosidase inhibitors for the treatment of Gaucher disease.Pharmacological chaperones for enzyme enhancement therapy in genetic diseases.The multivalent effect in glycosidase inhibition: a new, rapidly emerging topic in glycoscience.Emerging therapeutic targets for Gaucher disease.sp2-Iminosugar O-, S-, and N-glycosides as conformational mimics of α-linked disaccharides; implications for glycosidase inhibition.Targeting the Autophagy/Lysosomal Degradation Pathway in Parkinson's Disease.Recent advances and novel treatments for sphingolipidoses.Parkinson's disease: acid-glucocerebrosidase activity and alpha-synuclein clearance.Glycomimetic-based pharmacological chaperones for lysosomal storage disorders: lessons from Gaucher, GM1-gangliosidosis and Fabry diseases.New Directions in Gaucher Disease.Small molecules as therapeutic agents for inborn errors of metabolism.Glucosylceramide mimics: highly potent GCase inhibitors and selective pharmacological chaperones for mutations associated with types 1 and 2 Gaucher disease.Bicyclic derivatives of L-idonojirimycin as pharmacological chaperones for neuronopathic forms of Gaucher disease.Induced Pluripotent Stem Cell Modeling of Gaucher's Disease: What Have We Learned?Structural basis of pharmacological chaperoning for human β-galactosidase.Stabilization of Glucocerebrosidase by Active Site Occupancy.Biochemical phenotype of a common disease-causing mutation and a possible therapeutic approach for the phosphomannomutase 2-associated disorder of glycosylationAmbroxol as a pharmacological chaperone for mutant glucocerebrosidase.A systematic investigation of iminosugar click clusters as pharmacological chaperones for the treatment of Gaucher disease.New insights into the pharmacological chaperone activity of c2-substituted glucoimidazoles for the treatment of Gaucher disease.Accessing 2-substituted piperidine iminosugars by organometallic addition/intramolecular reductive amination: aldehyde vs. nitrone route.Glucocerebrosidase inhibitors: future drugs for the treatment of Gaucher disease?Targeted delivery of pharmacological chaperones for Gaucher disease to macrophages by a mannosylated cyclodextrin carrier.Pharmacotherapy of Gaucher Disease: Current and Future Options.
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Pharmacological chaperone therapy for Gaucher disease: a patent review.
description
article científic
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article scientifique
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articolo scientifico
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artigo científico
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bilimsel makale
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scientific article published on 04 April 2011
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vedecký článok
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vetenskaplig artikel
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videnskabelig artikel
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vědecký článek
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name
Pharmacological chaperone therapy for Gaucher disease: a patent review.
@en
Pharmacological chaperone therapy for Gaucher disease: a patent review.
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type
label
Pharmacological chaperone therapy for Gaucher disease: a patent review.
@en
Pharmacological chaperone therapy for Gaucher disease: a patent review.
@nl
prefLabel
Pharmacological chaperone therapy for Gaucher disease: a patent review.
@en
Pharmacological chaperone therapy for Gaucher disease: a patent review.
@nl
P2860
P50
P1476
Pharmacological chaperone therapy for Gaucher disease: a patent review
@en
P2093
José M García Fernández
P2860
P304
P356
10.1517/13543776.2011.569162
P407
P577
2011-04-04T00:00:00Z