The role of the iminosugar N-butyldeoxynojirimycin (miglustat) in the management of type I (non-neuronopathic) Gaucher disease: a position statement
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Enzyme replacement and substrate reduction therapy for Gaucher diseaseEnzyme replacement and substrate reduction therapy for Gaucher diseaseRescue of functional delF508-CFTR channels in cystic fibrosis epithelial cells by the alpha-glucosidase inhibitor miglustatUpdate on treatment of lysosomal storage diseasesImiglucerase in the treatment of Gaucher disease: a history and perspectiveProfile of eliglustat tartrate in the management of Gaucher diseaseClinical manifestations and management of Gaucher diseaseImiglucerase in the management of Gaucher disease type 1: an evidence-based review of its place in therapyNew therapeutic approaches for Krabbe disease: The potential of pharmacological chaperonesImiglucerase and its use for the treatment of Gaucher's diseaseSubstrate reduction augments the efficacy of enzyme therapy in a mouse model of Fabry diseaseEffective gene therapy in an authentic model of Tay-Sachs-related diseasesBiomarkers for lysosomal storage disorders: identification and application as exemplified by chitotriosidase in Gaucher disease.Gaucher disease: unmet treatment needs.Hemorrhagic aspects of Gaucher disease.A double-blind, randomized, placebo-controlled trial studying the effects of Saccharomyces boulardii on the gastrointestinal tolerability, safety, and pharmacokinetics of miglustat.Identification and characterization of ambroxol as an enzyme enhancement agent for Gaucher diseaseDrug repositioning for enzyme modulator based on human metabolite-likeness.The development and use of small molecule inhibitors of glycosphingolipid metabolism for lysosomal storage diseases.Skeletal improvement in patients with Gaucher disease type 1: a phase 2 trial of oral eliglustat.The European Gaucher Alliance: a survey of member patient organisations' activities, healthcare environments and concerns.Gene silencing of EXTL2 and EXTL3 as a substrate deprivation therapy for heparan sulphate storing mucopolysaccharidoses.Malignancies and monoclonal gammopathy in Gaucher disease; a systematic review of the literature.Antisense Oligonucleotide-mediated Suppression of Muscle Glycogen Synthase 1 Synthesis as an Approach for Substrate Reduction Therapy of Pompe DiseaseGaucher disease: clinical profile and therapeutic developments.Gaucher disease: pathological mechanisms and modern management.Miglustat has no apparent effect on spermatogenesis in normal men.Gaucher disease: new developments in treatment and etiology.Biomarkers in the diagnosis of lysosomal storage disorders: proteins, lipids, and inhibodies.Shaping the sperm head: an ER enzyme leaves its mark.A phase 2 multi-center, open-label, switch-over trial to evaluate the safety and efficacy of Abcertin® in patients with type 1 Gaucher disease.Carbohydrate-based therapeutics.Glycosphingolipid synthesis inhibition limits osteoclast activation and myeloma bone disease.An evidence-based review of the potential benefits of taliglucerase alfa in the treatment of patients with Gaucher diseaseRecommendations for the management of the haematological and onco-haematological aspects of Gaucher disease.New developments in treating glycosphingolipid storage diseases.Genetic diseases of sphingolipid metabolism: pathological mechanisms and therapeutic options.Pharmacotherapeutic strategies using small molecules for the treatment of glycolipid lysosomal storage disorders.Review of miglustat for clinical management in Gaucher disease type 1.Isofagomine- and 2,5-anhydro-2,5-imino-D-glucitol-based glucocerebrosidase pharmacological chaperones for Gaucher disease intervention.
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P2860
The role of the iminosugar N-butyldeoxynojirimycin (miglustat) in the management of type I (non-neuronopathic) Gaucher disease: a position statement
description
2003 nî lūn-bûn
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2003 թուականին հրատարակուած գիտական յօդուած
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2003 թվականին հրատարակված գիտական հոդված
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2003年の論文
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2003年論文
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2003年論文
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2003年論文
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2003年論文
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2003年論文
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2003年论文
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name
The role of the iminosugar N-b ...... disease: a position statement
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The role of the iminosugar N-b ...... disease: a position statement
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The role of the iminosugar N-b ...... disease: a position statement
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label
The role of the iminosugar N-b ...... disease: a position statement
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The role of the iminosugar N-b ...... disease: a position statement
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The role of the iminosugar N-b ...... disease: a position statement
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The role of the iminosugar N-b ...... disease: a position statement
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The role of the iminosugar N-b ...... disease: a position statement
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The role of the iminosugar N-b ...... disease: a position statement
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The role of the iminosugar N-b ...... disease: a position statement
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Advisory Council to the European Working Group on Gaucher Disease
G M Pastores
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10.1023/A:1025902113005
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2003-01-01T00:00:00Z
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1025781123