Guidance on the use of miglustat for treating patients with type 1 Gaucher disease.
about
The iminosugar isofagomine increases the activity of N370S mutant acid beta-glucosidase in Gaucher fibroblasts by several mechanismsDrug treatment of inborn errors of metabolism: a systematic reviewImiglucerase and its use for the treatment of Gaucher's diseaseIn vivo and ex vivo evaluation of L-type calcium channel blockers on acid beta-glucosidase in Gaucher disease mouse modelsEffect of oral eliglustat on splenomegaly in patients with Gaucher disease type 1: the ENGAGE randomized clinical trial.Case report of unexpected gastrointestinal involvement in type 1 Gaucher disease: comparison of eliglustat tartrate treatment and enzyme replacement therapyCarbohydrate mimetics and scaffolds: sweet spots in medicinal chemistry.Isofagomine in vivo effects in a neuronopathic Gaucher disease mouse.Multi-system disorders of glycosphingolipid and ganglioside metabolismELIGLUSTAT TARTRATE: Glucosylceramide Synthase Inhibitor Treatment of Type 1 Gaucher Disease.Gaucher's disease and cancer: a sphingolipid perspectiveGaucher disease: clinical profile and therapeutic developments.Modest phenotypic improvements in ASA-deficient mice with only one UDP-galactose:ceramide-galactosyltransferase gene.Drug discovery from natural sources.Property-based design of a glucosylceramide synthase inhibitor that reduces glucosylceramide in the brain.Selective action of the iminosugar isofagomine, a pharmacological chaperone for mutant forms of acid-beta-glucosidase.Carbohydrates in diversity-oriented synthesis: challenges and opportunities.Evaluation of miglustat as maintenance therapy after enzyme therapy in adults with stable type 1 Gaucher disease: a prospective, open-label non-inferiority studyBone turnover markers in patients with type 1 Gaucher disease.Pharmacotherapeutic strategies using small molecules for the treatment of glycolipid lysosomal storage disorders.Complementation of a pathogenic IFNGR2 misfolding mutation with modifiers of N-glycosylation.Glucocerebroside: an evolutionary advantage for patients with Gaucher disease and a new immunomodulatory agent.Secondary alterations of sphingolipid metabolism in lysosomal storage diseases.Discovery and design of carbohydrate-based therapeutics.Putative biological mechanisms of efficiency of substrate reduction therapies for mucopolysaccharidoses.Lysosomal storage disorders: A review of the musculoskeletal features.New insights into the pharmacological chaperone activity of c2-substituted glucoimidazoles for the treatment of Gaucher disease.
P2860
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P2860
Guidance on the use of miglustat for treating patients with type 1 Gaucher disease.
description
2005 nî lūn-bûn
@nan
2005 թուականի Նոյեմբերին հրատարակուած գիտական յօդուած
@hyw
2005 թվականի նոյեմբերին հրատարակված գիտական հոդված
@hy
2005年の論文
@ja
2005年論文
@yue
2005年論文
@zh-hant
2005年論文
@zh-hk
2005年論文
@zh-mo
2005年論文
@zh-tw
2005年论文
@wuu
name
Guidance on the use of miglustat for treating patients with type 1 Gaucher disease.
@ast
Guidance on the use of miglustat for treating patients with type 1 Gaucher disease.
@en
type
label
Guidance on the use of miglustat for treating patients with type 1 Gaucher disease.
@ast
Guidance on the use of miglustat for treating patients with type 1 Gaucher disease.
@en
prefLabel
Guidance on the use of miglustat for treating patients with type 1 Gaucher disease.
@ast
Guidance on the use of miglustat for treating patients with type 1 Gaucher disease.
@en
P2093
P356
P1476
Guidance on the use of miglustat for treating patients with type 1 Gaucher disease.
@en
P2093
Gregory A Grabowski
Henry J Mankin
Joel Charrow
John A Barranger
Neal J Weinreb
Pramod Mistry
P304
P356
10.1002/AJH.20504
P577
2005-11-01T00:00:00Z