An open-label, noncomparative study of miglustat in type I Gaucher disease: efficacy and tolerability over 24 months of treatment.
about
The iminosugar isofagomine increases the activity of N370S mutant acid beta-glucosidase in Gaucher fibroblasts by several mechanismsDysphagia as a risk factor for mortality in Niemann-Pick disease type C: systematic literature review and evidence from studies with miglustatDrug treatment of inborn errors of metabolism: a systematic reviewEffects of pH and Iminosugar Pharmacological Chaperones on Lysosomal Glycosidase Structure and StabilitySubstrate reduction augments the efficacy of enzyme therapy in a mouse model of Fabry diseaseRandomized, controlled trial of miglustat in Gaucher's disease type 3.An overview on bone manifestations in Gaucher disease.Gastrointestinal disturbances and their management in miglustat-treated patients.A double-blind, randomized, placebo-controlled trial studying the effects of Saccharomyces boulardii on the gastrointestinal tolerability, safety, and pharmacokinetics of miglustat.Imaging of Gaucher disease.Therapeutic approaches for lysosomal storage diseasesGaucher disease: clinical profile and therapeutic developments.Miglustat has no apparent effect on spermatogenesis in normal men.Substrate deprivation therapy: a new hope for patients suffering from neuronopathic forms of inherited lysosomal storage diseases.N-butyldeoxynojirimycin causes weight loss as a result of appetite suppression in lean and obese mice.Gaucher disease: new developments in treatment and etiology.Modest phenotypic improvements in ASA-deficient mice with only one UDP-galactose:ceramide-galactosyltransferase gene.Drug discovery from natural sources.Selective action of the iminosugar isofagomine, a pharmacological chaperone for mutant forms of acid-beta-glucosidase.Activities of genes controlling sphingolipid metabolism in human fibroblasts treated with flavonoids.Evaluation of miglustat as maintenance therapy after enzyme therapy in adults with stable type 1 Gaucher disease: a prospective, open-label non-inferiority studyPharmacotherapeutic strategies using small molecules for the treatment of glycolipid lysosomal storage disorders.Review of miglustat for clinical management in Gaucher disease type 1.Improved management of lysosomal glucosylceramide levels in a mouse model of type 1 Gaucher disease using enzyme and substrate reduction therapyPrediction of hand function after occupational hand injury by evaluation of initial anatomical severity.Targeting the bacteria-host interface: strategies in anti-adhesion therapy.Substrate reduction therapy.Enzyme Replacement Therapy in a Patient with Gaucher Disease Type III: A Paradigmatic Case Showing Severe Adverse Reactions Started a Long Time After the Beginning of TreatmentHuman chitinases and chitinase-like proteins as indicators for inflammation and cancerNew therapies in the management of Niemann-Pick type C disease: clinical utility of miglustatReal-world clinical experience with long-term miglustat maintenance therapy in type 1 Gaucher disease: the ZAGAL project.Treating lysosomal storage diseases with pharmacological chaperones: from concept to clinics.Pharmacological small molecules for the treatment of lysosomal storage disorders.Putative biological mechanisms of efficiency of substrate reduction therapies for mucopolysaccharidoses.Attitudes of Individuals with Gaucher Disease toward Substrate Reduction Therapies.Isofagomine induced stabilization of glucocerebrosidase.Dietary modifications in patients receiving miglustat.Miglustat in late-onset Tay-Sachs disease: a 12-month, randomized, controlled clinical study with 24 months of extended treatment.Gaucher's disease.Advances in male contraception
P2860
Q24673451-E6AC71DB-BB9C-4776-9AE5-C67A3A68362CQ26825102-5A654DDA-0B4D-4119-AD4D-6E490AB119E0Q26864796-1416B316-5237-4206-B581-73BF4BAF1D33Q27654912-D26D0316-80B0-4F2E-9832-D968453817F6Q28748170-DB90E198-2E2F-4E06-B55E-E94EC11C6E03Q30485096-1D8C7F4F-5358-46F2-8244-3F59B2576572Q33393590-7A45EF0A-5BAB-4C89-8735-5CC522BE9F46Q33451316-2284BF70-E71A-4CEA-8D89-9773610DD092Q33465330-43A3236A-1175-4B2F-800A-01CC9B2374EBQ34251797-7930E6C1-B7DD-4F1C-B3A0-4609C140DA2EQ34311420-2AD0C495-A263-4E2F-963A-06F93F164367Q34443517-4D5C26B9-3EA6-48AC-8D5C-4834B1B782F1Q34577027-345822D1-78FF-4234-9D57-793E892FD9A0Q34584825-2D0CCD80-C870-4ACD-B9F1-87E7457298A5Q34587013-6C462873-1774-40C6-80D2-C37142F5C98EQ34593914-B5B256C6-7019-4916-8A70-0E895CF914BEQ35024890-C08D81D1-7CE2-4C85-AA2E-9E2CA3E1F822Q35594873-DB582C57-B85C-4D4A-B247-8E4CE5A0B6AEQ35844420-CF4EE4C4-B5E1-45D8-B980-63224EBDB2C5Q36030017-89381CB7-11E3-4FB9-813D-85DFA8B1CCB3Q36556379-19033EF3-ECC0-435E-9018-F25F095624EEQ36739788-21EB2622-05C7-4A9D-8CD3-7E244950E261Q36823551-B0BA9D74-1F49-4E45-8ACB-357951E15501Q36932659-F7984759-11AB-4D84-B7F7-8E00642567A5Q36939541-8B097D93-91C0-44BA-9485-CECDAF9C1277Q37009022-14721B69-7038-4DBB-8355-6945A3D66BEBQ37109614-B5E42A3B-93E1-4240-B86F-C3A800F60A73Q37127644-453865B2-12C7-42CC-B62B-F2138FC4B6AFQ37280948-E0884F2B-9102-4568-9E45-B2F10099CDD0Q37429819-CBC8E100-30D2-4CC8-B2F5-86D5BE7B873EQ37470861-3DF2FA79-4308-4865-8733-B9BD4DE1F10CQ37668227-CEB86CDA-366A-40FC-AE0B-0FCD15698DABQ37799818-9E8B0E3F-9FC5-44D6-98AE-2A77B9E622E1Q38040710-A74838D8-595F-4AC4-9708-33FB20786B9EQ38627286-B21C4CA2-E29D-4DE9-A154-4B40BA69B100Q39927661-E62BFFDA-A450-40DB-B4E6-FB3DB98F3AB3Q44805716-E68D2CA6-B50E-4ED3-B71E-0FD4E8BE314FQ46060912-3C27BC9F-B761-45FC-AA0D-7459A936D2C4Q55619200-8A0706D5-4C51-4C7C-AEEF-BF7F94B0A828Q56679619-5D746CBD-4DBB-45DF-8BAB-AF38482A6697
P2860
An open-label, noncomparative study of miglustat in type I Gaucher disease: efficacy and tolerability over 24 months of treatment.
description
2005 nî lūn-bûn
@nan
2005 թուականի Օգոստոսին հրատարակուած գիտական յօդուած
@hyw
2005 թվականի օգոստոսին հրատարակված գիտական հոդված
@hy
2005年の論文
@ja
2005年学术文章
@wuu
2005年学术文章
@zh-cn
2005年学术文章
@zh-hans
2005年学术文章
@zh-my
2005年学术文章
@zh-sg
2005年學術文章
@yue
name
An open-label, noncomparative ...... y over 24 months of treatment.
@ast
An open-label, noncomparative ...... y over 24 months of treatment.
@en
type
label
An open-label, noncomparative ...... y over 24 months of treatment.
@ast
An open-label, noncomparative ...... y over 24 months of treatment.
@en
prefLabel
An open-label, noncomparative ...... y over 24 months of treatment.
@ast
An open-label, noncomparative ...... y over 24 months of treatment.
@en
P2093
P1476
An open-label, noncomparative ...... y over 24 months of treatment.
@en
P2093
Edwin H Kolodny
Gregory M Pastores
Natalie L Barnett
P304
P356
10.1016/J.CLINTHERA.2005.08.004
P577
2005-08-01T00:00:00Z