Randomized, controlled trial of miglustat in Gaucher's disease type 3. - Wikidata - wikimedia - TriplyDB

Randomized, controlled trial of miglustat in Gaucher's disease type 3.

Randomized, controlled trial of miglustat in Gaucher's disease type 3.

http://www.wikidata.org/entity/Q30485096

about

Enzyme replacement and substrate reduction therapy for Gaucher disease Enzyme replacement and substrate reduction therapy for Gaucher disease Hematopoietic stem cell transplantation for Gaucher disease Hematopoietic stem cell transplantation for Gaucher disease Less Is More: Substrate Reduction Therapy for Lysosomal Storage Disorders Profile of eliglustat tartrate in the management of Gaucher disease Eliglustat tartrate for the treatment of adults with type 1 Gaucher disease The clinical management of Type 2 Gaucher disease Iminosugar-based inhibitors of glucosylceramide synthase increase brain glycosphingolipids and survival in a mouse model of Sandhoff disease High throughput screening for small molecule therapy for Gaucher disease using patient tissue as the source of mutant glucocerebrosidase Central ocular motor disorders, including gaze palsy and nystagmus.The saccadic and neurological deficits in type 3 Gaucher disease.Characteristics of type I Gaucher disease associated with persistent thrombocytopenia after treatment with imiglucerase for 4-5 years.Gastrointestinal disturbances and their management in miglustat-treated patients.Induced pluripotent stem cell models of lysosomal storage disorders.The pharmacological chaperone isofagomine increases the activity of the Gaucher disease L444P mutant form of beta-glucosidase.Multi-system disorders of glycosphingolipid and ganglioside metabolism Development of targeted therapies for Parkinson's disease and related synucleinopathies Imaging of Gaucher disease.Discovery, structure-activity relationship, and biological evaluation of noninhibitory small molecule chaperones of glucocerebrosidase Therapeutic approaches for lysosomal storage diseases Gaucher disease and its treatment options.Gaucher disease: clinical profile and therapeutic developments.Enzyme replacement therapy and beyond-in memoriam Roscoe O. Brady, M.D. (1923-2016).Identification and characterization of pharmacological chaperones to correct enzyme deficiencies in lysosomal storage disorders.Identification of a biomarker in cerebrospinal fluid for neuronopathic forms of Gaucher disease.Pressure for drug development in lysosomal storage disorders - a quantitative analysis thirty years beyond the US orphan drug act Pharmacological Chaperones and Coenzyme Q10 Treatment Improves Mutant β-Glucocerebrosidase Activity and Mitochondrial Function in Neuronopathic Forms of Gaucher Disease.Lung lysed: A case of Gaucher disease with pulmonary involvement Ambroxol chaperone therapy for neuronopathic Gaucher disease: A pilot study.Successful therapy for protein-losing enteropathy caused by chronic neuronopathic Gaucher disease.Biochemical response to substrate reduction therapy versus enzyme replacement therapy in Gaucher disease type 1 patients CNS-accessible Inhibitor of Glucosylceramide Synthase for Substrate Reduction Therapy of Neuronopathic Gaucher Disease.Review of the safety and efficacy of imiglucerase treatment of Gaucher disease.Combination therapy in a patient with chronic neuronopathic Gaucher disease: a case report Progress and potential of non-inhibitory small molecule chaperones for the treatment of Gaucher disease and its implications for Parkinson disease.Emerging drugs for lysosomal storage diseases.Pharmacological small molecules for the treatment of lysosomal storage disorders.The pathophysiology of GD - current understanding and rationale for existing and emerging therapeutic approaches.Neuropathic Gaucher disease.

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P2860

Randomized, controlled trial of miglustat in Gaucher's disease type 3.

http://www.wikidata.org/entity/Q30485096

description

2008 nî lūn-bûn

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2008 թուականի Նոյեմբերին հրատարակուած գիտական յօդուած

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2008 թվականի նոյեմբերին հրատարակված գիտական հոդված

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2008年の論文

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2008年論文

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2008年論文

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2008年論文

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2008年論文

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2008年論文

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2008年论文

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name

Randomized, controlled trial of miglustat in Gaucher's disease type 3.

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Randomized, controlled trial of miglustat in Gaucher's disease type 3.

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type

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Randomized, controlled trial of miglustat in Gaucher's disease type 3.

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Randomized, controlled trial of miglustat in Gaucher's disease type 3.

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Randomized, controlled trial of miglustat in Gaucher's disease type 3.

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Randomized, controlled trial of miglustat in Gaucher's disease type 3.

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P1433

Annals of Neurology

P1476

Randomized, controlled trial of miglustat in Gaucher's disease type 3

@en

P2093

Ashok Vellodi

http://www.w3.org/2001/XMLSchema#string

Catherine DeVile

http://www.w3.org/2001/XMLSchema#string

Chris Harris

http://www.w3.org/2001/XMLSchema#string

Edmond J Fitzgibbon

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Elin H Davies

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Ivo N van Schaik

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Margaret Timmons

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Raphael Schiffmann

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William Benko

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P2860

The Gaucher registry: demographics and disease characteristics of 1698 patients with Gaucher disease.

An open-label, noncomparative study of miglustat in type I Gaucher disease: efficacy and tolerability over 24 months of treatment.

Miglustat for treatment of Niemann-Pick C disease: a randomised controlled study.

N-butyldeoxynojirimycin is a novel inhibitor of glycolipid biosynthesis.

Ten years' experience of enzyme infusion therapy of Norrbottnian (type 3) Gaucher disease.

Neuronopathic forms of Gaucher's disease.

Sustained therapeutic effects of oral miglustat (Zavesca, N-butyldeoxynojirimycin, OGT 918) in type I Gaucher disease.

Low-dose N-butyldeoxynojirimycin (OGT 918) for type I Gaucher disease.

Decreased bone density in splenectomized Gaucher patients receiving enzyme replacement therapy.

Pulmonary involvement in type 1 Gaucher disease: functional and exercise findings in patients with and without clinical interstitial lung disease.

P304

514-522

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scholarly article

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10.1002/ANA.21491

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5

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64

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2008-11-01T00:00:00Z

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23642717

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19067373

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2605167

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