Oral maintenance clinical trial with miglustat for type I Gaucher disease: switch from or combination with intravenous enzyme replacement.
about
Enzyme replacement and substrate reduction therapy for Gaucher diseaseCommon G102S polymorphism in chitotriosidase differentially affects activity towards 4-methylumbelliferyl substratesDiagnostic workup and management of patients with suspected Niemann-Pick type C diseaseImiglucerase and its use for the treatment of Gaucher's diseaseGastrointestinal 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.Multi-system disorders of glycosphingolipid and ganglioside metabolismImaging of Gaucher disease.Hepatobiliary quiz-10 (2014)Biomarkers in the diagnosis of lysosomal storage disorders: proteins, lipids, and inhibodies.Identification of potent and selective glucosylceramide synthase inhibitors from a library of N-alkylated iminosugars.Synthesis and Evaluation of Hybrid Structures Composed of Two Glucosylceramide Synthase Inhibitors.Observational cohort study of the natural history of Niemann-Pick disease type C in the UK: a 5-year update from the UK clinical database.Evaluation of miglustat as maintenance therapy after enzyme therapy in adults with stable type 1 Gaucher disease: a prospective, open-label non-inferiority studyBiochemical response to substrate reduction therapy versus enzyme replacement therapy in Gaucher disease type 1 patientsReview 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 therapySubstrate 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 TreatmentReview of the safety and efficacy of imiglucerase treatment of Gaucher disease.Successful switch from enzyme replacement therapy to miglustat in an adult patient with type 1 Gaucher disease: a case report.New 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.Determining the Role of the Aromatic Ring of N-Arylmethyl ent-conduramine F-1 in their Interactions with α-Glucosidases by Saturation Transfer Difference NMR Spectroscopy Experiments.Pharmacological small molecules for the treatment of lysosomal storage disorders.Gaucher disease and bone manifestations.Management of bone disease in Gaucher disease type 1: clinical practice.Attitudes of Individuals with Gaucher Disease toward Substrate Reduction Therapies.Combination Therapies for Lysosomal Storage Diseases: A Complex Answer to a Simple Problem.Modulation of glycogen synthesis by RNA interference: towards a new therapeutic approach for glycogenosis type II.Results from a 9-year Intensive Safety Surveillance Scheme (IS(3) ) in miglustat (Zavesca(®) )-treated patients.Synthesis and evaluation of eight- and four-membered iminosugar analogues as inhibitors of testicular ceramide-specific glucosyltransferase, testicular β-glucosidase 2, and other glycosidases.Restoration of muscle functionality by genetic suppression of glycogen synthesis in a murine model of Pompe disease.Combined miglustat and enzyme replacement therapy in two patients with type 1 Gaucher disease: two case reports.Gaucher disease
P2860
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P2860
Oral maintenance clinical trial with miglustat for type I Gaucher disease: switch from or combination with intravenous enzyme replacement.
description
2007 nî lūn-bûn
@nan
2007 թուականի Յուլիսին հրատարակուած գիտական յօդուած
@hyw
2007 թվականի հուլիսին հրատարակված գիտական հոդված
@hy
2007年の論文
@ja
2007年論文
@yue
2007年論文
@zh-hant
2007年論文
@zh-hk
2007年論文
@zh-mo
2007年論文
@zh-tw
2007年论文
@wuu
name
Oral maintenance clinical tria ...... ntravenous enzyme replacement.
@ast
Oral maintenance clinical tria ...... ntravenous enzyme replacement.
@en
Oral maintenance clinical tria ...... ntravenous enzyme replacement.
@nl
type
label
Oral maintenance clinical tria ...... ntravenous enzyme replacement.
@ast
Oral maintenance clinical tria ...... ntravenous enzyme replacement.
@en
Oral maintenance clinical tria ...... ntravenous enzyme replacement.
@nl
prefLabel
Oral maintenance clinical tria ...... ntravenous enzyme replacement.
@ast
Oral maintenance clinical tria ...... ntravenous enzyme replacement.
@en
Oral maintenance clinical tria ...... ntravenous enzyme replacement.
@nl
P2093
P1433
P1476
Oral maintenance clinical tria ...... intravenous enzyme replacement
@en
P2093
Altoon Dweck
Ari Zimran
Deborah Elstein
Drorit Attias
Gheona Altarescu
Irith Hadas-Halpern
Shoshana Zevin
Sonja van Weely
P304
P356
10.1182/BLOOD-2007-02-075960
P407
P50
P577
2007-07-03T00:00:00Z