Title: N-glycosylation determines the abundance of the transient receptor potential channel TRPP2.
Journal: The Journal of biological chemistry20140523
Title: Supersite of immune vulnerability on the glycosylated face of HIV-1 envelope glycoprotein gp120.
Journal: Nature structural & molecular biology20130701
Title: Miglustat-induced intestinal carbohydrate malabsorption is due to the inhibition of α-glucosidases, but not β-galactosidases.
Journal: Journal of inherited metabolic disease20121101
Title: Oxidative stress in Niemann-Pick type C patients: a protective role of N-butyl-deoxynojirimycin therapy.
Journal: International journal of developmental neuroscience : the official journal of the International Society for Developmental Neuroscience20121001
Title: Initiation and discontinuation of substrate inhibitor treatment in patients with Niemann-Pick type C disease.
Journal: Gene20120910
Title: Treatable metabolic psychoses that go undetected: what Niemann-Pick type C can teach us.
Journal: International journal of psychiatry in clinical practice20120901
Title: Miglustat as a therapeutic agent: prospects and caveats.
Journal: Journal of medical genetics20120901
Title: Recommendations for the diagnosis and management of Niemann-Pick disease type C: an update.
Journal: Molecular genetics and metabolism20120701
Title: Early miglustat therapy in infantile Niemann-Pick disease type C.
Journal: Pediatric neurology20120701
Title: Unexpected cure from cutaneous leukocytoclastic vasculitis in a patient treated with N-butyldeoxynojirimycin (miglustat) for Gaucher disease.
Journal: Advances in medical sciences20120601
Title: Discovery and characterization of an inhibitor of glucosylceramide synthase.
Journal: Journal of medicinal chemistry20120510
Title: A randomized placebo-controlled trial of miglustat in cystic fibrosis based on nasal potential difference.
Journal: Journal of cystic fibrosis : official journal of the European Cystic Fibrosis Society20120501
Title: Miglustat improves purkinje cell survival and alters microglial phenotype in feline Niemann-Pick disease type C.
Journal: Journal of neuropathology and experimental neurology20120501
Title: Iminosugar-based inhibitors of glucosylceramide synthase prolong survival but paradoxically increase brain glucosylceramide levels in Niemann-Pick C mice.
Journal: Molecular genetics and metabolism20120401
Title: Substrate reduction therapy with miglustat for type 1 Gaucher disease: a retrospective analysis from a single institution.
Journal: Upsala journal of medical sciences20120301
Title: Validated LC-MS/MS method for the quantitative determination of the glucosylceramide synthase inhibitor miglustat in mouse plasma and human plasma and its application to a pharmacokinetic study.
Journal: Journal of pharmaceutical and biomedical analysis20120205
Title: Extended remission of B-cell lymphoma with monoclonal gammopathy in a patient with type 1 Gaucher disease treated with enzyme replacement therapy.
Journal: Blood cells, molecules & diseases20120115
Title: High throughput screening for small molecule therapy for Gaucher disease using patient tissue as the source of mutant glucocerebrosidase.
Journal: PloS one20120101
Title: Gaucher disease and the synucleinopathies: refining the relationship.
Journal: Orphanet journal of rare diseases20120101
Title: Mapping the genetic and clinical characteristics of Gaucher disease in the Iberian Peninsula.
Journal: Orphanet journal of rare diseases20120101
Title: Imiglucerase in the treatment of Gaucher disease: a history and perspective.
Journal: Drug design, development and therapy20120101
Title: An evidence-based review of the potential benefits of taliglucerase alfa in the treatment of patients with Gaucher disease.
Journal: Core evidence20120101
Title: Miglustat therapy in the French cohort of paediatric patients with Niemann-Pick disease type C.
Journal: Orphanet journal of rare diseases20120101
Title: Therapeutic strategies for Gaucher disease: miglustat (NB-DNJ) as a pharmacological chaperone for glucocerebrosidase and the different thermostability of velaglucerase alfa and imiglucerase.
Journal: Molecular pharmaceutics20111205
Title: Gaucher disease due to saposin C deficiency, previously described as non-neuronopathic form--no positive effects after 2-years of miglustat therapy.
Journal: Molecular genetics and metabolism20111201
Title: Endoplasmic reticulum stress induces autophagy through activation of p38 MAPK in fibroblasts from Pompe disease patients carrying c.546G>T mutation.
Journal: Molecular genetics and metabolism20111201
Title: Translating clinical findings into knowledge in drug safety evaluation--drug induced liver injury prediction system (DILIps).
Journal: PLoS computational biology20111201
Title: Evaluation of miglustat treatment in patients with type III mucopolysaccharidosis: a randomized, double-blind, placebo-controlled study.
Journal: The Journal of pediatrics20111101
Title: Modulators of sphingolipid metabolism reduce lung inflammation.
Journal: American journal of respiratory cell and molecular biology20111001
Title: Glucosylceramide synthase inhibitor PDMP sensitizes chronic myeloid leukemia T315I mutant to Bcr-Abl inhibitor and cooperatively induces glycogen synthase kinase-3-regulated apoptosis.
Journal: FASEB journal : official publication of the Federation of American Societies for Experimental Biology20111001
Title: Gastrointestinal disturbances and their management in miglustat-treated patients.
Journal: Journal of inherited metabolic disease20111001
Title: Fusion failure of dense-cored proacrosomal vesicles in an inducible mouse model of male infertility.
Journal: Cell and tissue research20111001
Title: [Report of attendance at the Parseghian Scientific Conference for Niemann-Pick Type C (NP-C) Research, South Bend, Indiana: in search of new therapeutic possibilities for a very rare illness].
Journal: Kinderkrankenschwester : Organ der Sektion Kinderkrankenpflege20111001
Title: Synthesis and α-Glucosidase II inhibitory activity of valienamine pseudodisaccharides relevant to N-glycan biosynthesis.
Journal: Bioorganic & medicinal chemistry letters20110915
Title: Iminosugars as therapeutic agents: recent advances and promising trends.
Journal: Future medicinal chemistry20110901
Title: [Guidelines for type 1 Gaucher's disease].
Journal: Medicina clinica20110901
Title: New agents and approaches to treatment in Niemann-Pick type C disease.
Journal: Current pharmaceutical biotechnology20110601
Title: The many faces of the adamantyl group in drug design.
Journal: European journal of medicinal chemistry20110601
Title: Biomarkers in the diagnosis of lysosomal storage disorders: proteins, lipids, and inhibodies.
Journal: Journal of inherited metabolic disease20110601
Title: Synthesis of N-alkylated noeurostegines and evaluation of their potential as treatment for Gaucher's disease.
Journal: Bioorganic & medicinal chemistry letters20110301
Title: The videofluoroscopic swallowing study shows a sustained improvement of dysphagia in children with Niemann-Pick disease type C after therapy with miglustat.
Journal: American journal of medical genetics. Part A20110301
Title: Gamma-secretase-dependent amyloid-beta is increased in Niemann-Pick type C: a cross-sectional study.
Journal: Neurology20110125
Title: Evaluation of Spanish Gaucher disease patients after a 6-month imiglucerase shortage.
Journal: Blood cells, molecules & diseases20110115
Title: Treatment of cataplexy in Niemann-Pick disease type C with the use of miglustat.
Journal: European journal of paediatric neurology : EJPN : official journal of the European Paediatric Neurology Society20110101
Title: Influence of caloric restriction on constitutive expression of NF-κB in an experimental mouse astrocytoma.
Journal: PloS one20110101
Title: Bergamot (Citrus bergamia Risso) fruit extracts and identified components alter expression of interleukin 8 gene in cystic fibrosis bronchial epithelial cell lines.
Journal: BMC biochemistry20110101
Title: Limitations of drug registries to evaluate orphan medicinal products for the treatment of lysosomal storage disorders.
Journal: Orphanet journal of rare diseases20110101
Title: Isofagomine in vivo effects in a neuronopathic Gaucher disease mouse.
Journal: PloS one20110101
Title: Pricing and reimbursement of orphan drugs: the need for more transparency.
Journal: Orphanet journal of rare diseases20110101
Title: The potential investment impact of improved access to accelerated approval on the development of treatments for low prevalence rare diseases.
Journal: Orphanet journal of rare diseases20110101
Title: Iminosugar-based inhibitors of glucosylceramide synthase increase brain glycosphingolipids and survival in a mouse model of Sandhoff disease.
Journal: PloS one20110101
Title: Gaucher disease and cancer: concept and controversy.
Journal: International journal of cell biology20110101
Title: The saccadic and neurological deficits in type 3 Gaucher disease.
Journal: PloS one20110101
Title: Stimulation of Wild-Type, F508del- and G551D-CFTR Chloride Channels by Non-Toxic Modified pyrrolo[2,3-b]pyrazine Derivatives.
Journal: Frontiers in pharmacology20110101
Title: Gaucher's disease.
Journal: Indian journal of endocrinology and metabolism20110101
Title: Unusual presentation of adult Gaucher's disease: A long and difficult road to diagnosis.
Journal: Indian journal of endocrinology and metabolism20110101
Title: CFTR and Ca Signaling in Cystic Fibrosis.
Journal: Frontiers in pharmacology20110101
Title: Protein replacement therapy partially corrects the cholesterol-storage phenotype in a mouse model of Niemann-Pick type C2 disease.
Journal: PloS one20110101
Title: Corneal alterations during combined therapy with cyclodextrin/allopregnanolone and miglustat in a knock-out mouse model of NPC1 disease.
Journal: PloS one20110101
Title: Splenomegaly, cardiomegaly, and osteoporosis in a child with Gaucher disease.
Journal: Case reports in pediatrics20110101
Title: The pathophysiology of GD - current understanding and rationale for existing and emerging therapeutic approaches.
Journal: Wiener medizinische Wochenschrift (1946)20101201
Title: How adhesion/growth-regulatory galectins-1 and -3 attain cell specificity: case study defining their target on neuroblastoma cells (SK-N-MC) and marked affinity regulation by affecting microdomain organization of the membrane.
Journal: IUBMB life20100801
Title: Click chemistry approach to new N-substituted aminocyclitols as potential pharmacological chaperones for Gaucher disease.
Journal: Journal of medicinal chemistry20100722
Title: C terminus of nucleotide binding domain 1 contains critical features for cystic fibrosis transmembrane conductance regulator trafficking and activation.
Journal: The Journal of biological chemistry20100716
Title: Improved management of lysosomal glucosylceramide levels in a mouse model of type 1 Gaucher disease using enzyme and substrate reduction therapy.
Journal: Journal of inherited metabolic disease20100601
Title: Restricted ketogenic diet enhances the therapeutic action of N-butyldeoxynojirimycin towards brain GM2 accumulation in adult Sandhoff disease mice.
Journal: Journal of neurochemistry20100601
Title: [Clinical diagnosis of the adult form of Niemann-Pick type C disease].
Journal: Archives de pediatrie : organe officiel de la Societe francaise de pediatrie20100601
Title: [Therapies in the Niemann-Pick type C disease].
Journal: Archives de pediatrie : organe officiel de la Societe francaise de pediatrie20100601
Title: Changes in the atherogenic profile of patients with type 1 Gaucher disease after miglustat therapy.
Journal: Atherosclerosis20100401
Title: Miglustat in adult and juvenile patients with Niemann-Pick disease type C: long-term data from a clinical trial.
Journal: Molecular genetics and metabolism20100401
Title: Clinical experience with miglustat therapy in pediatric patients with Niemann-Pick disease type C: a case series.
Journal: Molecular genetics and metabolism20100401
Title: Long-term miglustat therapy in children with Niemann-Pick disease type C.
Journal: Journal of child neurology20100301
Title: Eye movement and diffusion tensor imaging analysis of treatment effects in a Niemann-Pick Type C patient.
Journal: Molecular genetics and metabolism20100301
Title: Dual-action lipophilic iminosugar improves glycemic control in obese rodents by reduction of visceral glycosphingolipids and buffering of carbohydrate assimilation.
Journal: Journal of medicinal chemistry20100128
Title: Force majeure: therapeutic measures in response to restricted supply of imiglucerase (Cerezyme) for patients with Gaucher disease.
Journal: Blood cells, molecules & diseases20100115
Title: Gateways to clinical trials.
Journal: Methods and findings in experimental and clinical pharmacology20100101
Title: QSAR Studies on andrographolide derivatives as α-glucosidase inhibitors.
Journal: International journal of molecular sciences20100101
Title: Niemann-Pick disease type C.
Journal: Orphanet journal of rare diseases20100101
Title: Expression of wild-type CFTR suppresses NF-kappaB-driven inflammatory signalling.
Journal: PloS one20100101
Title: Oxidative stress in Niemann-Pick disease, type C.
Journal: Molecular genetics and metabolism20100101
Title: Optimal therapy in Gaucher disease.
Journal: Therapeutics and clinical risk management20100101
Title: Bone events and evolution of biologic markers in Gaucher disease before and during treatment.
Journal: Arthritis research & therapy20100101
Title: Substrate reduction augments the efficacy of enzyme therapy in a mouse model of Fabry disease.
Journal: PloS one20100101
Title: Fabry disease - current treatment and new drug development.
Journal: Current chemical genomics20100101
Title: Eye movement impairment recovery in a Gaucher patient treated with miglustat.
Journal: Neurology research international20100101
Title: Gaucher disease: clinical profile and therapeutic developments.
Journal: Biologics : targets & therapy20100101
Title: Glycolipid-Dependent, Protease Sensitive Internalization of Pseudomonas aeruginosa Into Cultured Human Respiratory Epithelial Cells.
Journal: The open microbiology journal20100101
Title: Real-world clinical experience with long-term miglustat maintenance therapy in type 1 Gaucher disease: the ZAGAL project.
Journal: Haematologica20091201
Title: Synthesis and evaluation of D-gluco-pyranocyclopropyl amines as potential glucosidase inhibitors.
Journal: Bioorganic & medicinal chemistry letters20091201
Title: [Treatment of bone involvement in Gaucher diseases with imiglucerase combined with miglustat].
Journal: Presse medicale (Paris, France : 1983)20091201
Title: Miglustat in patients with Niemann-Pick disease Type C (NP-C): a multicenter observational retrospective cohort study.
Journal: Molecular genetics and metabolism20091101
Title: [Gaucher's disease in Rennes University hospital: a 10-year retrospective study].
Journal: La Revue de medecine interne20091001
Title: Gateways to clinical trials.
Journal: Methods and findings in experimental and clinical pharmacology20091001
Title: Miglustat (Zavesca) in type 1 Gaucher disease: 5-year results of a post-authorisation safety surveillance programme.
Journal: Pharmacoepidemiology and drug safety20090901
Title: A cystic fibrosis respiratory epithelial cell chronically treated by miglustat acquires a non-cystic fibrosis-like phenotype.
Journal: American journal of respiratory cell and molecular biology20090801
Title: Pharmacokinetics, safety and tolerability of miglustat in the treatment of pediatric patients with GM2 gangliosidosis.
Journal: Molecular genetics and metabolism20090801
Title: The pharmacological chaperone N-butyldeoxynojirimycin enhances enzyme replacement therapy in Pompe disease fibroblasts.
Journal: Molecular therapy : the journal of the American Society of Gene Therapy20090601
Title: Airway delivery of low-dose miglustat normalizes nasal potential difference in F508del cystic fibrosis mice.
Journal: American journal of respiratory and critical care medicine20090601
Title: Miglustat in late-onset Tay-Sachs disease: a 12-month, randomized, controlled clinical study with 24 months of extended treatment.
Journal: Genetics in medicine : official journal of the American College of Medical Genetics20090601
Title: [Recent therapeutics for Fabry disease].
Journal: Nihon Naika Gakkai zasshi. The Journal of the Japanese Society of Internal Medicine20090410
Title: Gateways to clinical trials.
Journal: Methods and findings in experimental and clinical pharmacology20090401
Title: 24 month-treatment with miglustat of three patients with Niemann-Pick disease type C: follow up using brain spectroscopy.
Journal: Molecular genetics and metabolism20090201
Title: Rapid quantification of miglustat in human plasma and cerebrospinal fluid by liquid chromatography coupled with tandem mass spectrometry.
Journal: Journal of chromatography. B, Analytical technologies in the biomedical and life sciences20090115
Title: Promising results of the chaperone effect caused by imino sugars and aminocyclitol derivatives on mutant glucocerebrosidases causing Gaucher disease.
Journal: Blood cells, molecules & diseases20090101
Title: Goal-oriented therapy with miglustat in Gaucher disease.
Journal: Current medical research and opinion20090101
Title: Mechanisms of the noxious inflammatory cycle in cystic fibrosis.
Journal: Respiratory research20090101
Title: Substrate reduction therapy in juvenile GM2 gangliosidosis.
Journal: Molecular genetics and metabolism20090101
Title: Chronic cyclodextrin treatment of murine Niemann-Pick C disease ameliorates neuronal cholesterol and glycosphingolipid storage and disease progression.
Journal: PloS one20090101
Title: Review of the safety and efficacy of imiglucerase treatment of Gaucher disease.
Journal: Biologics : targets & therapy20090101
Title: In vivo and ex vivo evaluation of L-type calcium channel blockers on acid beta-glucosidase in Gaucher disease mouse models.
Journal: PloS one20090101
Title: New therapies in the management of Niemann-Pick type C disease: clinical utility of miglustat.
Journal: Therapeutics and clinical risk management20090101
Title: Treatment of a child diagnosed with Niemann-Pick disease type C with miglustat: a case report in Brazil.
Journal: Journal of inherited metabolic disease20081201
Title: Anti-inflammatory effect of miglustat in bronchial epithelial cells.
Journal: Journal of cystic fibrosis : official journal of the European Cystic Fibrosis Society20081101
Title: Randomized, controlled trial of miglustat in Gaucher's disease type 3.
Journal: Annals of neurology20081101
Title: Glycosphingolipid depletion in PC12 cells using iminosugars protects neuronal membranes from anti-ganglioside antibody mediated injury.
Journal: Journal of neuroimmunology20081015
Title: N-Butyldeoxynojirimycin is a broadly effective anti-HIV therapy significantly enhanced by targeted liposome delivery.
Journal: AIDS (London, England)20081001
Title: Neurocognitive testing in late-onset Tay-Sachs disease: a pilot study.
Journal: Journal of inherited metabolic disease20080801
Title: Changes of bone metabolism in seven patients with Gaucher disease treated consecutively with imiglucerase and miglustat.
Journal: Calcified tissue international20080701
Title: Immunoglobulin and free light chain abnormalities in Gaucher disease type I: data from an adult cohort of 63 patients and review of the literature.
Journal: Annals of hematology20080601
Title: Parallel improvement of sodium and chloride transport defects by miglustat (n-butyldeoxynojyrimicin) in cystic fibrosis epithelial cells.
Journal: The Journal of pharmacology and experimental therapeutics20080601
Title: Splenic nodules in paediatric Gaucher disease treated by enzyme replacement therapy.
Journal: Pediatric radiology20080601
Title: Beneficial effects of substrate reduction therapy in a mouse model of GM1 gangliosidosis.
Journal: Molecular genetics and metabolism20080601
Title: Differential sensitivity of mouse strains to an N-alkylated imino sugar: glycosphingolipid metabolism and acrosome formation.
Journal: Pharmacogenomics20080601
Title: Proteasome-dependent pharmacological rescue of cystic fibrosis transmembrane conductance regulator revealed by mutation of glycine 622.
Journal: The Journal of pharmacology and experimental therapeutics20080401
Title: Gaucher disease: improving management.
Journal: Acta paediatrica (Oslo, Norway : 1992)20080401
Title: Gaucher disease: unmet treatment needs.
Journal: Acta paediatrica (Oslo, Norway : 1992)20080401
Title: Substrate reduction therapy.
Journal: Acta paediatrica (Oslo, Norway : 1992)20080401
Title: Review of miglustat for clinical management in Gaucher disease type 1.
Journal: Therapeutics and clinical risk management20080401
Title: [New data on Gaucher's disease].
Journal: La Revue de medecine interne20080301
Title: Gaucher disease.
Journal: Joint, bone, spine : revue du rhumatisme20080301
Title: Recommendations for the assessment and monitoring of skeletal manifestations in children with Gaucher disease.
Journal: Skeletal radiology20080301
Title: New perspectives in non-hormonal male contraception.
Journal: Trends in endocrinology and metabolism: TEM20080301
Title: Calcium homeostasis is abnormal in cystic fibrosis airway epithelial cells but is normalized after rescue of F508del-CFTR.
Journal: Cell calcium20080201
Title: N-butyldeoxynojirimycin causes weight loss as a result of appetite suppression in lean and obese mice.
Journal: Diabetes, obesity & metabolism20080201
Title: Motion analysis of a child with Niemann-Pick disease type C treated with miglustat.
Journal: Movement disorders : official journal of the Movement Disorder Society20080101
Title: Gateways to clinical trials.
Journal: Methods and findings in experimental and clinical pharmacology20080101
Title: Differential effects of energy stress on AMPK phosphorylation and apoptosis in experimental brain tumor and normal brain.
Journal: Molecular cancer20080101
Title: Abnormal spatial diffusion of Ca2+ in F508del-CFTR airway epithelial cells.
Journal: Respiratory research20080101
Title: [Osteoarticular manifestations of Gaucher disease in adults: pathophysiology and treatment].
Journal: Presse medicale (Paris, France : 1983)20071201
Title: [Niemann-Pick diseases in adults].
Journal: La Revue de medecine interne20071201
Title: Accumulation of glucosylceramide in murine testis, caused by inhibition of beta-glucosidase 2: implications for spermatogenesis.
Journal: The Journal of biological chemistry20071109
Title: Treatment of Niemann-Pick disease type C in two children with miglustat: initial responses and maintenance of effects over 1 year.
Journal: Journal of inherited metabolic disease20071001
Title: Oral maintenance clinical trial with miglustat for type I Gaucher disease: switch from or combination with intravenous enzyme replacement.
Journal: Blood20071001
Title: Influence of food intake on the pharmacokinetics of miglustat, an inhibitor of glucosylceramide synthase.
Journal: Journal of clinical pharmacology20071001
Title: [Therapeutic objectives in Gaucher disease].
Journal: La Revue de medecine interne20071001
Title: [Osteoarticular manifestations of Gaucher disease in adults: pathophysiology and treatment].
Journal: La Revue de medecine interne20071001
Title: [Current development and usefulness of biomarkers for Gaucher disease follow up].
Journal: La Revue de medecine interne20071001
Title: [Organization of Gaucher disease management in France].
Journal: La Revue de medecine interne20071001
Title: Crystal structures of complexes of N-butyl- and N-nonyl-deoxynojirimycin bound to acid beta-glucosidase: insights into the mechanism of chemical chaperone action in Gaucher disease.
Journal: The Journal of biological chemistry20070928
Title: Recommendations for the management of the haematological and onco-haematological aspects of Gaucher disease.
Journal: British journal of haematology20070901
Title: Miglustat for treatment of Niemann-Pick C disease: a randomised controlled study.
Journal: The Lancet. Neurology20070901
Title: Effective treatment of an elderly patient with Gaucher's disease and Parkinsonism: a case report of 24 months' oral substrate reduction therapy with miglustat.
Journal: Parkinsonism & related disorders20070801
Title: Effect of miglustat on bone disease in adults with type 1 Gaucher disease: a pooled analysis of three multinational, open-label studies.
Journal: Clinical therapeutics20070801
Title: Neurologic improvement in a type 3 Gaucher disease patient treated with imiglucerase/miglustat combination.
Journal: Epilepsia20070701
Title: [Molecular pathogenesis and therapeutic targets of lysosomal diseases].
Journal: Seikagaku. The Journal of Japanese Biochemical Society20070701
Title: Differential alteration of lipid antigen presentation to NKT cells due to imbalances in lipid metabolism.
Journal: European journal of immunology20070601
Title: Selective action of the iminosugar isofagomine, a pharmacological chaperone for mutant forms of acid-beta-glucosidase.
Journal: Biochemical pharmacology20070501
Title: Miglustat has no apparent effect on spermatogenesis in normal men.
Journal: Human reproduction (Oxford, England)20070301
Title: [Is it possible to treat the brain? Recent advances in inherited neurodegenerative disorders].
Journal: No to hattatsu = Brain and development20070301
Title: The pharmacokinetics and tissue distribution of the glucosylceramide synthase inhibitor miglustat in the rat.
Journal: Xenobiotica; the fate of foreign compounds in biological systems20070301
Title: Imino sugars are potent agonists of the human glucose sensor SGLT3.
Journal: Molecular pharmacology20070201
Title: Severe endothelial dysfunction in the aorta of a mouse model of Fabry disease; partial prevention by N-butyldeoxynojirimycin treatment.
Journal: Journal of inherited metabolic disease20070201
Title: The sensitivity of murine spermiogenesis to miglustat is a quantitative trait: a pharmacogenetic study.
Journal: Reproductive biology and endocrinology : RB&E20070101
Title: [Iminosugars: current and future therapeutic applications].
Journal: Annales pharmaceutiques francaises20070101
Title: [Cost reduction in the treatment of Gaucher s disease].
Journal: Farmacia hospitalaria : organo oficial de expresion cientifica de la Sociedad Espanola de Farmacia Hospitalaria20070101
Title: Substrate deprivation therapy: a new hope for patients suffering from neuronopathic forms of inherited lysosomal storage diseases.
Journal: Journal of applied genetics20070101
Title: Human chitinases and chitinase-like proteins as indicators for inflammation and cancer.
Journal: Biomarker insights20070101
Title: Clinical experience with substrate reduction therapy.
Journal: European journal of internal medicine20061101
Title: Synthesis and evaluation of glycosidase inhibitory activity of N-butyl 1-deoxy-D-gluco-homonojirimycin and N-butyl 1-deoxy-L-ido-homonojirimycin.
Journal: Bioorganic & medicinal chemistry20060815
Title: Inhibition of hybrid- and complex-type glycosylation reveals the presence of the GlcNAc transferase I-independent fucosylation pathway.
Journal: Glycobiology20060801
Title: Gangliosides do not affect ABC transporter function in human neuroblastoma cells.
Journal: Journal of lipid research20060601
Title: Long-term non-hormonal male contraception in mice using N-butyldeoxynojirimycin.
Journal: Human reproduction (Oxford, England)20060501
Title: Short-term effect of miglustat in every day clinical use in treatment-naïve or previously treated patients with type 1 Gaucher's disease.
Journal: Haematologica20060501
Title: D-threo-1-phenyl-2-decanoylamino-3-morpholino-1-propanol alters cellular cholesterol homeostasis by modulating the endosome lipid domains.
Journal: Biochemistry20060411
Title: Rescue of functional delF508-CFTR channels in cystic fibrosis epithelial cells by the alpha-glucosidase inhibitor miglustat.
Journal: FEBS letters20060403
Title: Gaucher disease: multiple lessons from a single gene disorder.
Journal: Acta paediatrica (Oslo, Norway : 1992). Supplement20060401
Title: [Clinical study of the French cohort of Gaucher disease patients].
Journal: La Revue de medecine interne20060301
Title: Therapeutic goals in Gaucher disease.
Journal: La Revue de medecine interne20060301
Title: Substrate reduction therapy in the infantile form of Tay-Sachs disease.
Journal: Neurology20060124
Title: Miglustat: substrate reduction therapy for glycosphingolipid lysosomal storage disorders.
Journal: Drugs of today (Barcelona, Spain : 1998)20060101
Title: Gateways to clinical trials.
Journal: Methods and findings in experimental and clinical pharmacology20060101
Title: Emerging strategies for the treatment of hereditary metabolic storage disorders.
Journal: Rejuvenation research20060101
Title: Substrate reduction therapy of glycosphingolipid storage disorders.
Journal: Journal of inherited metabolic disease20060101
Title: Modest phenotypic improvements in ASA-deficient mice with only one UDP-galactose:ceramide-galactosyltransferase gene.
Journal: Lipids in health and disease20060101
Title: Miglustat: substrate reduction therapy for lysosomal storage disorders associated with primary central nervous system involvement.
Journal: Recent patents on CNS drug discovery20060101
Title: Guidance on the use of miglustat for treating patients with type 1 Gaucher disease.
Journal: American journal of hematology20051101
Title: Imino sugar inhibitors for treating the lysosomal glycosphingolipidoses.
Journal: Glycobiology20051001
Title: Miglustat: new drug. In type 1 Gaucher's disease : a slight benefit after imiglucerase therapy.
Journal: Prescrire international20051001
Title: An open-label, noncomparative study of miglustat in type I Gaucher disease: efficacy and tolerability over 24 months of treatment.
Journal: Clinical therapeutics20050801
Title: Glycosphingolipid accumulation inhibits cholesterol efflux via the ABCA1/apolipoprotein A-I pathway: 1-phenyl-2-decanoylamino-3-morpholino-1-propanol is a novel cholesterol efflux accelerator.
Journal: The Journal of biological chemistry20050701
Title: Alkylated imino sugars, reversible male infertility-inducing agents, do not affect the genetic integrity of male mouse germ cells during short-term treatment despite induction of sperm deformities.
Journal: Biology of reproduction20050401
Title: Substrate reduction therapy for lysosomal storage diseases.
Journal: Acta paediatrica (Oslo, Norway : 1992). Supplement20050301
Title: Computerized cognitive testing in patients with type I Gaucher disease: effects of enzyme replacement and substrate reduction.
Journal: Genetics in medicine : official journal of the American College of Medical Genetics20050201
Title: Miglustat (NB-DNJ) works as a chaperone for mutated acid beta-glucosidase in cells transfected with several Gaucher disease mutations.
Journal: Blood cells, molecules & diseases20050101
Title: Absence of pathogenic mitochondrial DNA mutations in mouse brain tumors.
Journal: BMC cancer20050101
Title: C-glycosides and aza-C-glycosides as potential glycosidase and glycosyltransferase inhibitors.
Journal: Current topics in medicinal chemistry20050101
Title: Synthesis and glycosidase inhibitory activity of new hexa-substituted C8-glycomimetics.
Journal: Beilstein journal of organic chemistry20050101
Title: Alpha-1-C-alkyl-1-deoxynojirimycin derivatives as potent and selective inhibitors of intestinal isomaltase: remarkable effect of the alkyl chain length on glycosidase inhibitory profile.
Journal: Bioorganic & medicinal chemistry letters20041220
Title: Inhibition of alpha-glucosidases I and II increases the cell surface expression of functional class A macrophage scavenger receptor (SR-A) by extending its half-life.
Journal: The Journal of biological chemistry20040917
Title: Treatment with miglustat reverses the lipid-trafficking defect in Niemann-Pick disease type C.
Journal: Neurobiology of disease20040801
Title: Ceramide synthesis correlates with the posttranscriptional regulation of the sterol-regulatory element-binding protein.
Journal: Arteriosclerosis, thrombosis, and vascular biology20040501
Title: Action of celgosivir (6 O-butanoyl castanospermine) against the pestivirus BVDV: implications for the treatment of hepatitis C.
Journal: Antiviral chemistry & chemotherapy20040501
Title: [Type 1 Gaucher's disease in the adult. Nutritional management during initiation of treatment with miglustat].
Journal: Presse medicale (Paris, France : 1983)20040410
Title: New drugs of 2003.
Journal: Journal of the American Pharmacists Association : JAPhA20040101
Title: Sustained therapeutic effects of oral miglustat (Zavesca, N-butyldeoxynojirimycin, OGT 918) in type I Gaucher disease.
Journal: Journal of inherited metabolic disease20040101
Title: N-butyldeoxynojirimycin inhibits murine melanoma cell ganglioside metabolism and delays tumor onset.
Journal: Cancer letters20031110
Title: Inhibition of calcium uptake via the sarco/endoplasmic reticulum Ca2+-ATPase in a mouse model of Sandhoff disease and prevention by treatment with N-butyldeoxynojirimycin.
Journal: The Journal of biological chemistry20030808
Title: Epitope-dependent blocking of the angiotensin-converting enzyme dimerization by monoclonal antibodies to the N-terminal domain of ACE: possible link of ACE dimerization and shedding from the cell surface.
Journal: Biochemistry20030617
Title: Substrate reduction therapy: clinical evaluation in type 1 Gaucher disease.
Journal: Philosophical transactions of the Royal Society of London. Series B, Biological sciences20030529
Title: Gaucher disease and the clinical experience with substrate reduction therapy.
Journal: Philosophical transactions of the Royal Society of London. Series B, Biological sciences20030529
Title: Gateways to clinical trials.
Journal: Methods and findings in experimental and clinical pharmacology20030501
Title: Central nervous system inflammation is a hallmark of pathogenesis in mouse models of GM1 and GM2 gangliosidosis.
Journal: Brain : a journal of neurology20030401
Title: Miglustat. Oxford GlycoSciences/Actelion.
Journal: Current opinion in investigational drugs (London, England : 2000)20030401
Title: [Drug for Gaucher disease also works as sperm inhibitor. Is the 'pill' for the man on its way?].
Journal: MMW Fortschritte der Medizin20030213
Title: Substrate reduction therapy: miglustat as a remedy for symptomatic patients with Gaucher disease type 1.
Journal: Expert opinion on investigational drugs20030201
Title: Glycolipid depletion in antimicrobial therapy.
Journal: Molecular microbiology20030101
Title: The role of the iminosugar N-butyldeoxynojirimycin (miglustat) in the management of type I (non-neuronopathic) Gaucher disease: a position statement.
Journal: Journal of inherited metabolic disease20030101
Title: Miglustat.
Journal: Drugs20030101
Title: New therapeutics for the treatment of glycosphingolipid lysosomal storage diseases.
Journal: Advances in experimental medicine and biology20030101
Title: Calnexin, calreticulin, and ERp57 cooperate in disulfide bond formation in human CD1d heavy chain.
Journal: The Journal of biological chemistry20021122
Title: Glycosphingolipid lysosomal storage diseases: therapy and pathogenesis.
Journal: Neuropathology and applied neurobiology20021001
Title: The combination of interferon alpha-2b and n-butyl deoxynojirimycin has a greater than additive antiviral effect upon production of infectious bovine viral diarrhea virus (BVDV) in vitro: implications for hepatitis C virus (HCV) therapy.
Journal: Antiviral research20020901
Title: Imino sugars that are less toxic but more potent as antivirals, in vitro, compared with N-n-nonyl DNJ.
Journal: Antiviral chemistry & chemotherapy20020901
Title: Dietary restriction reduces angiogenesis and growth in an orthotopic mouse brain tumour model.
Journal: British journal of cancer20020520
Title: pH-sensitive liposomes are efficient carriers for endoplasmic reticulum-targeted drugs in mouse melanoma cells.
Journal: Biochemical and biophysical research communications20020510
Title: Inhibition of host ER glucosidase activity prevents Golgi processing of virion-associated bovine viral diarrhea virus E2 glycoproteins and reduces infectivity of secreted virions.
Journal: Virology20020330
Title: Low-dose N-butyldeoxynojirimycin (OGT 918) for type I Gaucher disease.
Journal: Blood cells, molecules & diseases20020101
Title: Regulation of apoptosis during neuronal differentiation by ceramide and b-series complex gangliosides.
Journal: The Journal of biological chemistry20011130
Title: Critical role for glycosphingolipids in Niemann-Pick disease type C.
Journal: Current biology : CB20010821
Title: N -butyldeoxynojirimycin reduces growth and ganglioside content of experimental mouse brain tumours.
Journal: British journal of cancer20010401
Title: Substrate reduction therapy for glycosphingolipid storage disorders.
Journal: Expert opinion on investigational drugs20010301
Title: Enhanced survival in Sandhoff disease mice receiving a combination of substrate deprivation therapy and bone marrow transplantation.
Journal: Blood20010101
Title: Imino sugars inhibit the formation and secretion of bovine viral diarrhea virus, a pestivirus model of hepatitis C virus: implications for the development of broad spectrum anti-hepatitis virus agents.
Journal: Proceedings of the National Academy of Sciences of the United States of America19991012
Title: Differential effects of glycolipid biosynthesis inhibitors on ceramide-induced cell death in neuroblastoma cells.
Journal: Journal of neurochemistry19990301
Title: Synthesis of alpha-glucosidase I inhibitors showing antiviral (HIV-1) and immunosuppressive activity.
Journal: The Journal of pharmacy and pharmacology19960201
Title: N-alkylated nitrogen-in-the-ring sugars: conformational basis of inhibition of glycosidases and HIV-1 replication.
Journal: Journal of medicinal chemistry19950623
Title: Inhibition of alpha-glucosidase I of the glycoprotein-processing enzymes by 6-O-butanoyl castanospermine (MDL 28,574) and its consequences in human immunodeficiency virus-infected T cells.
Journal: Antimicrobial agents and chemotherapy19940801
Title: The safety and efficacy of combination N-butyl-deoxynojirimycin (SC-48334) and zidovudine in patients with HIV-1 infection and 200-500 CD4 cells/mm3.
Journal: Journal of acquired immune deficiency syndromes19940201
Title: 6-0-butanoylcastanospermine (MDL 28,574) inhibits glycoprotein processing and the growth of HIVs.
Journal: AIDS (London, England)19910601
Title: Inhibition of HIV and SIV infectivity by blockade of alpha-glucosidase activity.
Journal: Virology19910301
Title: Inhibitory effect of novel 1-deoxynojirimycin derivatives on HIV-1 replication.
Journal: AIDS (London, England)19901001
Title: Attenuation of HIV-1 infectivity by an inhibitor of oligosaccharide processing.
Journal: AIDS research and human retroviruses19900601
Title: Inhibition of HIV replication by amino-sugar derivatives.
Journal: FEBS letters19880912
Title: Abian, O., et al., Therapeutic strategies for Gaucher disease: miglustat (NB-DNJ) as a pharmacological chaperone for glucocerebrosidase and the different thermostability of velaglucerase alfa and imiglucerase. Mol Pharm, 2011. 8(6): p. 2390-7.
Title: van Giersbergen, P.L. and J. Dingemanse, Influence of food intake on the pharmacokinetics of miglustat, an inhibitor of glucosylceramide synthase. J Clin Pharmacol, 2007. 47(10): p. 1277-82.
