Glycomimetic-based pharmacological chaperones for lysosomal storage disorders: lessons from Gaucher, GM1-gangliosidosis and Fabry diseases

Influence of the configurational pattern of sp2-iminosugar pseudo N-, S-, O- and C-glycosides on their glycoside inhibitory and antitumor properties

(2016) Elena M. Sánchez-Fernández et al.   CARBOHYDRATE RESEARCH

Efficient stereoselective synthesis of 2-acetamido-1,2-dideoxyallonojirimycin (DAJNAc) and sp 2 -iminosugar conjugates: Novel hexosaminidase inhibitors with discrimination capabilities between the mature and precursor forms of the enzyme

(2016) Alex de la Fuente et al.   EUROPEAN JOURNAL OF MEDICINAL CHEMISTRY

Inhibitor versus chaperone behaviour of d -fagomine, DAB and LAB sp 2 -iminosugar conjugates against glycosidases: A structure–activity relationship study in Gaucher fibroblasts

(2016) Teresa Mena-Barragán et al.   EUROPEAN JOURNAL OF MEDICINAL CHEMISTRY

From blood–brain barrier to blood–brain interface: new opportunities for CNS drug delivery

(2016) William A. Banks   NATURE REVIEWS DRUG DISCOVERY

Docking study and biological evaluation of pyrrolidine-based iminosugars as pharmacological chaperones for Gaucher disease

(2016) Atsushi Kato et al.   ORGANIC & BIOMOLECULAR CHEMISTRY

Conformationally-locked C-glycosides: tuning aglycone interactions for optimal chaperone behaviour in Gaucher fibroblasts

(2016) C. D. Navo et al.   ORGANIC & BIOMOLECULAR CHEMISTRY

pH-Responsive Pharmacological Chaperones for Rescuing Mutant Glycosidases

(2015) Teresa Mena-Barragán et al.   ANGEWANDTE CHEMIE-INTERNATIONAL EDITION

Lysosomal Storage Diseases: From Pathophysiology to Therapy

(2015) Giancarlo Parenti et al.   Annual Review of Medicine

Glyco-engineering strategies for the development of therapeutic enzymes with improved efficacy for the treatment of lysosomal storage diseases

(2015) Doo-Byoung Oh   BMB Reports

Human Acid β-Glucosidase Inhibition by Carbohydrate Derived Iminosugars: Towards New Pharmacological Chaperones for Gaucher Disease

(2015) Camilla Parmeggiani et al.   CHEMBIOCHEM

N-Alkyl-, 1-C-Alkyl-, and 5-C-Alkyl-1,5-dideoxy-1,5-imino-(l)-ribitols as Galactosidase Inhibitors

(2015) Sophie Front et al.   ChemMedChem

Looking for protein stabilizing drugs with thermal shift assay

(2015) Giuseppina Andreotti et al.   Drug Testing and Analysis

Eliglustat: A Review in Gaucher Disease Type 1

(2015) Lesley J. Scott   DRUGS

Conformationally-locked N-glycosides: Exploiting long-range non-glycone interactions in the design of pharmacological chaperones for Gaucher disease

(2015) Javier Castilla et al.   EUROPEAN JOURNAL OF MEDICINAL CHEMISTRY

Chaperone therapy for Krabbe disease: potential for late-onset GALC mutations

(2015) Mohammad Arif Hossain et al.   JOURNAL OF HUMAN GENETICS

Fluorescence-Quenched Substrates for Live Cell Imaging of Human Glucocerebrosidase Activity

(2015) Anuj K. Yadav et al.   JOURNAL OF THE AMERICAN CHEMICAL SOCIETY

Coformulation of a Novel Human α -Galactosidase A With the Pharmacological Chaperone AT1001 Leads to Improved Substrate Reduction in Fabry Mice

(2015) Su Xu et al.   MOLECULAR THERAPY

Pharmacological Chaperone Therapy: Preclinical Development, Clinical Translation, and Prospects for the Treatment of Lysosomal Storage Disorders

(2015) Giancarlo Parenti et al.   MOLECULAR THERAPY

Drug repositioning can accelerate discovery of pharmacological chaperones

(2015) Bruno Hay Mele et al.   Orphanet Journal of Rare Diseases

Quality of life in patients with Fabry disease: a systematic review of the literature

(2015) Maarten Arends et al.   Orphanet Journal of Rare Diseases

Pressure for drug development in lysosomal storage disorders – a quantitative analysis thirty years beyond the US orphan drug act

(2015) Konstantin Mechler et al.   Orphanet Journal of Rare Diseases

Azasugar inhibitors as pharmacological chaperones for Krabbe disease

(2015) Chris H. Hill et al.   Chemical Science

Potent chemical chaperone compounds for GM1-gangliosidosis: N-substituted (+)-conduramine F-4 derivatives

(2015) Shinichi Kuno et al.   MedChemComm

Direct synthesis of imino-C-nucleoside analogues and other biologically active iminosugars

(2015) Milan Bergeron-Brlek et al.   Nature Communications

Antileishmanial activity of sp2-iminosugar derivatives

(2015) Elena M. Sánchez-Fernández et al.   RSC Advances

Pharmacological Chaperones and Coenzyme Q10 Treatment Improves Mutant β-Glucocerebrosidase Activity and Mitochondrial Function in Neuronopathic Forms of Gaucher Disease

(2015) Mario de la Mata et al.   Scientific Reports

Enzyme replacement therapy: lessons learned and emerging questions

(2015) Agnieszka Jurecka et al.   Expert Opinion on Orphan Drugs

Visualization of Active Glucocerebrosidase in Rodent Brain with High Spatial Resolution following In Situ Labeling with Fluorescent Activity Based Probes

(2015) Daniela Herrera Moro Chao et al.   PLoS One

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

(2015) David G. Warnock et al.   PLoS One

Molecular Basis of 1-Deoxygalactonojirimycin Arylthiourea Binding to Human α-Galactosidase A: Pharmacological Chaperoning Efficacy on Fabry Disease Mutants

(2014) Yi Yu et al.   ACS Chemical Biology

Docking and SAR studies of calystegines: Binding orientation and influence on pharmacological chaperone effects for Gaucher’s disease

(2014) Atsushi Kato et al.   BIOORGANIC & MEDICINAL CHEMISTRY

Correction of lysosomal dysfunction as a therapeutic strategy for neurodegenerative diseases

(2014) Robert E. Boyd et al.   BIOORGANIC & MEDICINAL CHEMISTRY LETTERS

Concise synthesis of C-1-cyano-iminosugars via a new Staudinger/aza Wittig/Strecker multicomponent reaction strategy

(2014) Manuel Zoidl et al.   BIOORGANIC & MEDICINAL CHEMISTRY LETTERS

The Multivalent Effect in Glycosidase Inhibition: A New, Rapidly Emerging Topic in Glycoscience

(2014) Philippe Compain et al.   CHEMBIOCHEM

Multivalent Inhibitors for Carbohydrate-Processing Enzymes: Beyond the “Lock-and-Key” Concept

(2014) Sébastien G. Gouin   CHEMISTRY-A EUROPEAN JOURNAL

Emerging therapeutic targets for Gaucher disease

(2014) Einat B Vitner et al.   EXPERT OPINION ON THERAPEUTIC TARGETS

Selective chaperone effect of aminocyclitol derivatives on G202R and other mutant glucocerebrosidases causing Gaucher disease

(2014) Jenny Serra-Vinardell et al.   INTERNATIONAL JOURNAL OF BIOCHEMISTRY & CELL BIOLOGY

Structural Basis of Pharmacological Chaperoning for Human β-Galactosidase

(2014) Hironori Suzuki et al.   JOURNAL OF BIOLOGICAL CHEMISTRY

The development and use of small molecule inhibitors of glycosphingolipid metabolism for lysosomal storage diseases

(2014) James A. Shayman et al.   JOURNAL OF LIPID RESEARCH

Synthesis of Multibranched Australine Derivatives from Reducing Castanospermine Analogues through the Amadori Rearrangement of gem-Diamine Intermediates: Selective Inhibitors of β-Glucosidase

(2014) Elena M. Sánchez-Fernández et al.   JOURNAL OF ORGANIC CHEMISTRY

Synthesis of 1,5-Dideoxy-1,5-iminoribitol C-Glycosides through a Nitrone–Olefin Cycloaddition Domino Strategy: Identification of Pharmacological Chaperones of Mutant Human Lysosomal β-Galactosidase

(2014) Aloysius Siriwardena et al.   JOURNAL OF ORGANIC CHEMISTRY

A Chaperone Enhances Blood α-Glucosidase Activity in Pompe Disease Patients Treated With Enzyme Replacement Therapy

(2014) Giancarlo Parenti et al.   MOLECULAR THERAPY

Sphingolipid lysosomal storage disorders

(2014) Frances M. Platt   NATURE

Disturbance of endoplasmic reticulum proteostasis in neurodegenerative diseases

(2014) Claudio Hetz et al.   NATURE REVIEWS NEUROSCIENCE

Targeted delivery of pharmacological chaperones for Gaucher disease to macrophages by a mannosylated cyclodextrin carrier

(2014) Julio Rodríguez-Lavado et al.   ORGANIC & BIOMOLECULAR CHEMISTRY

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

(2014) Richie Khanna et al.   PLoS One

Searching for Glycomimetics That Target Protein Misfolding in Rare Diseases: Successes, Failures, and Unexpected Progress Made in Organic Synthesis

(2014) Philippe Compain   SYNLETT

Rapid modifications of N-substitution in iminosugars: Development of new β-glucocerebrosidase inhibitors and pharmacological chaperones for Gaucher disease

(2013) Wei-Chieh Cheng et al.   BIOORGANIC & MEDICINAL CHEMISTRY

Bicyclic Derivatives ofL-Idonojirimycin as Pharmacological Chaperones for Neuronopathic Forms of Gaucher Disease

(2013) Pilar Alfonso et al.   CHEMBIOCHEM

A diversity oriented one-pot synthesis of novel iminosugar C-glycosides

(2013) Soundararasu Senthilkumar et al.   CHEMICAL COMMUNICATIONS

Recent developments in design and synthesis of bicyclic azasugars, carbasugars and related molecules as glycosidase inhibitors

(2013) Rima Lahiri et al.   CHEMICAL SOCIETY REVIEWS

Fullerene-sp2-Iminosugar Balls as Multimodal Ligands for Lectins and Glycosidases: A Mechanistic Hypothesis for the Inhibitory Multivalent Effect

(2013) Rocío Rísquez-Cuadro et al.   CHEMISTRY-A EUROPEAN JOURNAL

Enhancing Biodistribution of Therapeutic Enzymes In Vivo by Modulating Surface Coating and Concentration of ICAM-1-Targeted Nanocarriers

(2013) Janet Hsu et al.   Journal of Biomedical Nanotechnology

Treatment effect of coenzyme Q10 and an antioxidant cocktail in fibroblasts of patients with Sanfilippo disease

(2013) Leslie Matalonga et al.   JOURNAL OF INHERITED METABOLIC DISEASE

Pharmacological Chaperones as Therapeutics for Lysosomal Storage Diseases

(2013) Robert E. Boyd et al.   JOURNAL OF MEDICINAL CHEMISTRY

A Phase 2 study of migalastat hydrochloride in females with Fabry disease: Selection of population, safety and pharmacodynamic effects

(2013) R. Giugliani et al.   MOLECULAR GENETICS AND METABOLISM

A Bicyclic 1-Deoxygalactonojirimycin Derivative as a Novel Pharmacological Chaperone for GM1 Gangliosidosis

(2013) Tomoko Takai et al.   MOLECULAR THERAPY

Migalastat HCl Reduces Globotriaosylsphingosine (Lyso-Gb3) in Fabry Transgenic Mice and in the Plasma of Fabry Patients

(2013) Brandy Young-Gqamana et al.   PLoS One

New Castanospermine Glycoside Analogues Inhibit Breast Cancer Cell Proliferation and Induce Apoptosis without Affecting Normal Cells

(2013) Ghada Allan et al.   PLoS One

Stereoselective synthesis of 1-C-alkyl iminogalactitol derivatives, potential chaperones for galactosidase-linked LSDs: a real challenge

(2013) Anna Biela-Banaś et al.   TETRAHEDRON LETTERS

Adamantane substituted aminocyclitols as pharmacological chaperones for Gaucher disease

(2013) Ana Trapero et al.   MedChemComm

Pilot study using ambroxol as a pharmacological chaperone in type 1 Gaucher disease

(2012) Ari Zimran et al.   BLOOD CELLS MOLECULES AND DISEASES

Tuning glycosidase inhibition through aglycone interactions: pharmacological chaperones for Fabry disease and GM1 gangliosidosis

(2012) M. Aguilar-Moncayo et al.   CHEMICAL COMMUNICATIONS

sp2-Iminosugar O-, S-, and N-Glycosides as Conformational Mimics of α-Linked Disaccharides; Implications for Glycosidase Inhibition

(2012) Elena M. Sánchez-Fernández et al.   CHEMISTRY-A EUROPEAN JOURNAL

Systematic Structure-Activity Study on Potential Chaperone Lead Compounds for Acid α-Glucosidase

(2012) Chiara Bruckmann et al.   ChemMedChem

The Development of an Aza-C-Glycoside Library Based on a Tandem Staudinger/Aza-Wittig/Ugi Three-Component Reaction

(2012) Tom Wennekes et al.   EUROPEAN JOURNAL OF ORGANIC CHEMISTRY

Neuronopathic Gaucher's disease: induced pluripotent stem cells for disease modelling and testing chaperone activity of small compounds

(2012) Gustavo Tiscornia et al.   HUMAN MOLECULAR GENETICS

α-Galactosidase Aggregation Is a Determinant of Pharmacological Chaperone Efficacy on Fabry Disease Mutants

(2012) Aleksandra Siekierska et al.   JOURNAL OF BIOLOGICAL CHEMISTRY

Lysosomal storage disorders: The cellular impact of lysosomal dysfunction

(2012) Frances M. Platt et al.   JOURNAL OF CELL BIOLOGY

Conformationally-Locked N-Glycosides with Selective β-Glucosidase Inhibitory Activity: Identification of a New Non-Iminosugar-Type Pharmacological Chaperone for Gaucher Disease

(2012) Javier Castilla et al.   JOURNAL OF MEDICINAL CHEMISTRY

Potent Aminocyclitol Glucocerebrosidase Inhibitors are Subnanomolar Pharmacological Chaperones for Treating Gaucher Disease

(2012) Ana Trapero et al.   JOURNAL OF MEDICINAL CHEMISTRY

Rapid Assembly of a Library of Lipophilic Iminosugars via the Thiol–Ene Reaction Yields Promising Pharmacological Chaperones for the Treatment of Gaucher Disease

(2012) Ethan D. Goddard-Borger et al.   JOURNAL OF MEDICINAL CHEMISTRY

Therapeutic chaperone effect of N-Octyl 4-Epi-β-valienamine on murine GM1-gangliosidosis

(2012) Yoshiyuki Suzuki et al.   MOLECULAR GENETICS AND METABOLISM

The role of saposin C in Gaucher disease

(2012) Rafael J. Tamargo et al.   MOLECULAR GENETICS AND METABOLISM

Evaluation of N-nonyl-deoxygalactonojirimycin as a pharmacological chaperone for human GM1 gangliosidosis leads to identification of a feline model suitable for testing enzyme enhancement therapy

(2012) Brigitte A. Rigat et al.   MOLECULAR GENETICS AND METABOLISM

Co-administration With the Pharmacological Chaperone AT1001 Increases Recombinant Human α-Galactosidase A Tissue Uptake and Improves Substrate Reduction in Fabry Mice

(2012) Elfrida R Benjamin et al.   MOLECULAR THERAPY

Developing inhibitors of glycan processing enzymes as tools for enabling glycobiology

(2012) Tracey M Gloster et al.   Nature Chemical Biology

Synthesis of N-substituted ε-hexonolactams as pharmacological chaperones for the treatment of N370S mutant Gaucher disease

(2012) Guan-Nan Wang et al.   ORGANIC & BIOMOLECULAR CHEMISTRY

Safety and pharmacodynamic effects of a pharmacological chaperone on α-galactosidase A activity and globotriaosylceramide clearance in Fabry disease: report from two phase 2 clinical studies

(2012) Dominique P Germain et al.   Orphanet Journal of Rare Diseases

The Pharmacological Chaperone AT2220 Increases Recombinant Human Acid α-Glucosidase Uptake and Glycogen Reduction in a Mouse Model of Pompe Disease

(2012) Richie Khanna et al.   PLoS One

Pharmacological chaperones for human  -N-acetylgalactosaminidase

(2012) N. E. Clark et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Glycosidase inhibition by novel guanidinium and urea iminosugar derivatives

(2012) Raymond Kooij et al.   MedChemComm

Binding of 3,4,5,6-Tetrahydroxyazepanes to the Acid-β-glucosidase Active Site: Implications for Pharmacological Chaperone Design for Gaucher Disease

(2011) Susan D. Orwig et al.   BIOCHEMISTRY

Docking and SAR studies of d- and l-isofagomine isomers as human β-glucocerebrosidase inhibitors

(2011) Atsushi Kato et al.   BIOORGANIC & MEDICINAL CHEMISTRY

A fluorescent probe for GM1 gangliosidosis related β-galactosidase: N-(Dansylamino)hexylaminocarbonylpentyl-1,5-dideoxy-1,5-imino-d-galactitol

(2011) Richard F.G. Fröhlich et al.   BIOORGANIC & MEDICINAL CHEMISTRY LETTERS

Molecular basis of neurogenetic diseases

(2011) Yoshiyuki Suzuki   BRAIN & DEVELOPMENT

1-Deoxy-d-galactonojirimycins with dansyl capped N-substituents as β-galactosidase inhibitors and potential probes for GM1 gangliosidosis affected cell lines

(2011) Richard F.G. Fröhlich et al.   CARBOHYDRATE RESEARCH

Synthesis, Kinetic Evaluation and Cell-Based Analysis of C-Alkylated Isofagomines as Chaperones of β-Glucocerebrosidase

(2011) Tara Hill et al.   CHEMBIOCHEM

Recent applications of thiol–ene coupling as a click process for glycoconjugation

(2011) Alessandro Dondoni et al.   CHEMICAL SOCIETY REVIEWS

The Molecular Basis of Pharmacological Chaperoning in Human α-Galactosidase

(2011) Abigail I. Guce et al.   CHEMISTRY & BIOLOGY

Pharmacological chaperone therapy for Gaucher disease: a patent review

(2011) Juan M Benito et al.   EXPERT OPINION ON THERAPEUTIC PATENTS

Chemical chaperone therapy: chaperone effect on mutant enzyme and cellular pathophysiology in β-galactosidase deficiency

(2011) Katsumi Higaki et al.   HUMAN MUTATION

A pharmacogenetic approach to identify mutant forms of α-galactosidase a that respond to a pharmacological chaperone for Fabry disease

(2011) Xiaoyang Wu et al.   HUMAN MUTATION

Ex Vivoandin VivoEffects of Isofagomine on Acid β-Glucosidase Variants and Substrate Levels in Gaucher Disease

(2011) Ying Sun et al.   JOURNAL OF BIOLOGICAL CHEMISTRY

Crystal Structure of Human β-Galactosidase

(2011) Umeharu Ohto et al.   JOURNAL OF BIOLOGICAL CHEMISTRY

Fluorous iminoalditols act as effective pharmacological chaperones against gene products from GLB1 alleles causing GM1-gangliosidosis and Morquio B disease

(2011) Katrin M. Fantur et al.   JOURNAL OF INHERITED METABOLIC DISEASE

Lysosomal accumulation of Trk protein in brain of GM1-gangliosidosis mouse and its restoration by chemical chaperone

(2011) Ayumi Takamura et al.   JOURNAL OF NEUROCHEMISTRY

Amphiphilic 1-Deoxynojirimycin Derivatives through Click Strategies for Chemical Chaperoning in N370S Gaucher Cells

(2011) Jennifer D. Diot et al.   JOURNAL OF ORGANIC CHEMISTRY

Polyhydroxylated Bicyclic Isoureas and Guanidines Are Potent Glucocerebrosidase Inhibitors and Nanomolar Enzyme Activity Enhancers in Gaucher Cells

(2011) Ana Trapero et al.   JOURNAL OF THE AMERICAN CHEMICAL SOCIETY

Molecular mechanism for stabilization of a mutant α-galactosidase A involving M51I amino acid substitution by imino sugars

(2011) Takahiro Tsukimura et al.   MOLECULAR GENETICS AND METABOLISM

Bicyclic (galacto)nojirimycin analogues as glycosidase inhibitors: Effect of structural modifications in their pharmacological chaperone potential towards β-glucocerebrosidase

(2011) Matilde Aguilar-Moncayo et al.   ORGANIC & BIOMOLECULAR CHEMISTRY

Cyclodextrin-mediated crystallization of acid β-glucosidase in complex with amphiphilic bicyclic nojirimycin analogues

(2011) Boris Brumshtein et al.   ORGANIC & BIOMOLECULAR CHEMISTRY

Looking-Glass Synergistic Pharmacological Chaperones: DGJ and L-DGJ from the Enantiomers of Tagatose

(2011) Sarah F. Jenkinson et al.   ORGANIC LETTERS

Isofagomine In Vivo Effects in a Neuronopathic Gaucher Disease Mouse

(2011) Ying Sun et al.   PLoS One

The myo-1,2-Diaminocyclitol Scaffold Defines Potent Glucocerebrosidase Activators and Promising Pharmacological Chaperones for Gaucher Disease

(2011) Ana Trapero et al.   ACS Medicinal Chemistry Letters

Synthesis of lipophilic 1-deoxygalactonojirimycin derivatives as D-galactosidase inhibitors

(2010) Georg Schitter et al.   Beilstein Journal of Organic Chemistry

2,5-Dideoxy-2,5-imino-d-altritol as a new class of pharmacological chaperone for Fabry disease

(2010) Atsushi Kato et al.   BIOORGANIC & MEDICINAL CHEMISTRY

A Fluorescent sp2-Iminosugar With Pharmacological Chaperone Activity for Gaucher Disease: Synthesis and Intracellular Distribution Studies

(2010) Zhuo Luan  et al.   CHEMBIOCHEM

Fluorous Iminoalditols: A New Family of Glycosidase Inhibitors and Pharmacological Chaperones

(2010) Georg Schitter et al.   CHEMBIOCHEM

Synthesis of N-, S-, and C-glycoside castanospermine analogues with selective neutral α-glucosidase inhibitory activity as antitumour agents

(2010) Elena M. Sánchez-Fernández et al.   CHEMICAL COMMUNICATIONS

Iminosugars past, present and future: medicines for tomorrow

(2010) Graeme Horne et al.   DRUG DISCOVERY TODAY

Molecular Basis of Reduced Glucosylceramidase Activity in the Most Common Gaucher Disease Mutant, N370S

(2010) Marc N. Offman et al.   JOURNAL OF BIOLOGICAL CHEMISTRY

X-ray and Biochemical Analysis of N370S Mutant Human Acid β-Glucosidase

(2010) Ronnie R. Wei et al.   JOURNAL OF BIOLOGICAL CHEMISTRY

Chemical chaperone therapy: Luciferase assay for screening of β-galactosidase mutations

(2010) Linjing Li et al.   MOLECULAR GENETICS AND METABOLISM

DLHex-DGJ, a novel derivative of 1-deoxygalactonojirimycin with pharmacological chaperone activity in human GM1-gangliosidosis fibroblasts

(2010) Katrin Fantur et al.   MOLECULAR GENETICS AND METABOLISM

Ultrasensitive in situ visualization of active glucocerebrosidase molecules

(2010) Martin D Witte et al.   Nature Chemical Biology

Glycosphingolipids-Nature, Function, and Pharmacological Modulation

(2009) Tom Wennekes et al.   ANGEWANDTE CHEMIE-INTERNATIONAL EDITION

Effects of pH and Iminosugar Pharmacological Chaperones on Lysosomal Glycosidase Structure and Stability

(2009) Raquel L. Lieberman et al.   BIOCHEMISTRY

Promising results of the chaperone effect caused by iminosugars and aminocyclitol derivatives on mutant glucocerebrosidases causing Gaucher disease

(2009) Gessamí Sánchez-Ollé et al.   BLOOD CELLS MOLECULES AND DISEASES

Chaperone Activity of Bicyclic Nojirimycin Analogues for Gaucher Mutations in Comparison withN-(n-nonyl)Deoxynojirimycin

(2009) Zhuo Luan et al.   CHEMBIOCHEM

6-Amino-6-deoxy-5,6-di-N-(N′-octyliminomethylidene)nojirimycin: Synthesis, Biological Evaluation, and Crystal Structure in Complex with Acid β-Glucosidase

(2009) Boris Brumshtein et al.   CHEMBIOCHEM

Design, Synthesis, and Biological Evaluation of Enantiomeric β-N-Acetylhexosaminidase Inhibitors LABNAc and DABNAc as Potential Agents against Tay-Sachs and Sandhoff Disease

(2009) J. S. Shane Rountree et al.   ChemMedChem

Effects of a chemical chaperone on genetic mutations in α-galactosidase A in Korean patients with Fabry disease

(2009) Jung-Young Park et al.   EXPERIMENTAL AND MOLECULAR MEDICINE

The pharmacological chaperone 1-deoxygalactonojirimycin increases α-galactosidase A levels in Fabry patient cell lines

(2009) E. R. Benjamin et al.   JOURNAL OF INHERITED METABOLIC DISEASE

Rational Design and Synthesis of Highly Potent Pharmacological Chaperones for Treatment of N370S Mutant Gaucher Disease

(2009) Guan-Nan Wang et al.   JOURNAL OF MEDICINAL CHEMISTRY

Synthesis of Thiohydantoin-Castanospermine Glycomimetics as Glycosidase Inhibitors

(2009) Matilde Aguilar-Moncayo et al.   JOURNAL OF ORGANIC CHEMISTRY

The Pharmacological Chaperone 1-Deoxygalactonojirimycin Reduces Tissue Globotriaosylceramide Levels in a Mouse Model of Fabry Disease

(2009) Richie Khanna et al.   MOLECULAR THERAPY

From carbohydrate leads to glycomimetic drugs

(2009) Beat Ernst et al.   NATURE REVIEWS DRUG DISCOVERY

Multicenter Analysis of Glucocerebrosidase Mutations in Parkinson's Disease

(2009) E. Sidransky et al.   NEW ENGLAND JOURNAL OF MEDICINE

Glycosidase inhibition by ring-modified castanospermine analogues: tackling enzyme selectivity by inhibitor tailoring

(2009) Matilde Aguilar-Moncayo et al.   ORGANIC & BIOMOLECULAR CHEMISTRY

Synthesis and glycosidase inhibitory activity of noeurostegine—a new and potent inhibitor of β-glucoside hydrolases

(2009) Tina Secher Rasmussen et al.   ORGANIC & BIOMOLECULAR CHEMISTRY

Glycosidase inhibition: assessing mimicry of the transition state

(2009) Tracey M. Gloster et al.   ORGANIC & BIOMOLECULAR CHEMISTRY

Generalized Anomeric Effect ingem-Diamines: Stereoselective Synthesis of α-N-Linked Disaccharide Mimics

(2009) Elena M. Sánchez-Fernández et al.   ORGANIC LETTERS

Protein Misfolding as an Underlying Molecular Defect in Mucopolysaccharidosis III Type C

(2009) Matthew Feldhammer et al.   PLoS One

Tactics and strategies for the synthesis of iminosugar C-glycosides: a review

(2009) Philippe Compain et al.   TETRAHEDRON-ASYMMETRY

Substrate reduction therapy

(2008) Frances M Platt et al.   ACTA PAEDIATRICA

Synthesis and evaluation of sulfamide-type indolizidines as glycosidase inhibitors

(2008) Mahmoud Benltifa et al.   BIOORGANIC & MEDICINAL CHEMISTRY LETTERS

Molecular Basis for β-Glucosidase Inhibition by Ring-Modified Calystegine Analogues

(2008) Matilde Aguilar et al.   CHEMBIOCHEM

Current Developments in the Synthesis and Biological Activity of Aza-C-Nucleosides:Immucillins and Related Compounds

(2008) Pedro Merino et al.   CURRENT MEDICINAL CHEMISTRY

The potential action of galactose as a “chemical chaperone”: Increase of beta galactosidase activity in fibroblasts from an adult GM1-gangliosidosis patient

(2008) Anna Caciotti et al.   EUROPEAN JOURNAL OF PAEDIATRIC NEUROLOGY

Synthesis and Biological Evaluation of Guanidine-Type Iminosugars

(2008) Matilde Aguilar et al.   JOURNAL OF ORGANIC CHEMISTRY

GM1 gangliosidosis: Review of clinical, molecular, and therapeutic aspects

(2008) Nicola Brunetti-Pierri et al.   MOLECULAR GENETICS AND METABOLISM