Dose-response relationships for enzyme replacement therapy with imiglucerase/alglucerase in patients with Gaucher disease type 1. - Wikidata - awgldk - TriplyDB

Dose-response relationships for enzyme replacement therapy with imiglucerase/alglucerase in patients with Gaucher disease type 1.

Dose-response relationships for enzyme replacement therapy with imiglucerase/alglucerase in patients with Gaucher disease type 1.

http://www.wikidata.org/entity/Q37221766

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Enzyme replacement and substrate reduction therapy for Gaucher disease Therapeutic potential of intracerebroventricular replacement of modified human β-hexosaminidase B for GM2 gangliosidosis Imiglucerase in the treatment of Gaucher disease: a history and perspective Osteocyte Alterations Induce Osteoclastogenesis in an In Vitro Model of Gaucher Disease A Review of Gaucher Disease Pathophysiology, Clinical Presentation and Treatments A phase 2 study of eliglustat tartrate (Genz-112638), an oral substrate reduction therapy for Gaucher disease type 1.Thrombocytopenia and bleeding in dental procedures of patients with Gaucher disease.Characteristics of type I Gaucher disease associated with persistent thrombocytopenia after treatment with imiglucerase for 4-5 years.Long-term clinical outcomes in type 1 Gaucher disease following 10 years of imiglucerase treatment Achievement of therapeutic goals with low-dose imiglucerase in Gaucher disease: a single-center experience.Comparative therapeutic effects of velaglucerase alfa and imiglucerase in a Gaucher disease mouse model.Isofagomine in vivo effects in a neuronopathic Gaucher disease mouse.Modeling changes in biomarkers in Gaucher disease patients receiving enzyme replacement therapy using a pathophysiological model.The European Gaucher Alliance: a survey of member patient organisations' activities, healthcare environments and concerns.Bone events and evolution of biologic markers in Gaucher disease before and during treatment.Modelling Gaucher disease progression: long-term enzyme replacement therapy reduces the incidence of splenectomy and bone complications.A reappraisal of Gaucher disease-diagnosis and disease management algorithms.Limitations of drug registries to evaluate orphan medicinal products for the treatment of lysosomal storage disorders.Reducing selection bias in case-control studies from rare disease registries.Impact of imiglucerase supply constraint on the therapeutic management and course of disease in French patients with Gaucher disease type 1 Evaluation of disease burden and response to treatment in adults with type 1 Gaucher disease using a validated disease severity scoring system (DS3)Improved management of lysosomal glucosylceramide levels in a mouse model of type 1 Gaucher disease using enzyme and substrate reduction therapy CNS-accessible Inhibitor of Glucosylceramide Synthase for Substrate Reduction Therapy of Neuronopathic Gaucher Disease.Enzyme therapy for the treatment of type 1 Gaucher disease: clinical outcomes and dose - response relationships.Gaucher disease and bone manifestations.Innovative research methods for studying treatments for rare diseases: methodological review.Velaglucerase alfa in the treatment of Gaucher disease type 1 Long-term treatment outcomes in Gaucher disease.Modelling long-term evolution of chitotriosidase in non-neuronopathic Gaucher disease.Evaluation of a low dose, after a standard therapeutic dose, of agalsidase beta during enzyme replacement therapy in patients with Fabry disease.The history and accomplishments of the ICGG Gaucher registry.Gaucher disease: resetting the clinical and scientific agenda.Optimal therapy in Gaucher disease Characteristics of 26 patients with type 3 Gaucher disease: A descriptive analysis from the Gaucher Outcome Survey.Gaucher disease: spectrum of sonographic findings in the liver.Positive Effectiveness of Tafamidis in Delaying Disease Progression in Transthyretin Familial Amyloid Polyneuropathy up to 2 Years: An Analysis from the Transthyretin Amyloidosis Outcomes Survey (THAOS).

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Dose-response relationships for enzyme replacement therapy with imiglucerase/alglucerase in patients with Gaucher disease type 1.

http://www.wikidata.org/entity/Q37221766

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scientific article published on February 2009

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Genetics in Medicine

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Ari Zimran

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Carla E M Hollak

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Gregory A Grabowski

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J Alexander Cole

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Joel Charrow

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John Yee

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Katherine Kacena

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Lin Zhang

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Pramod K Mistry

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Stephan vom Dahl

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P2860

Gaucher disease: alendronate disodium improves bone mineral density in adults receiving enzyme therapy.

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

Individualization of long-term enzyme replacement therapy for Gaucher disease.

Adult-type Gaucher disease in children: genetics, clinical features and enzyme replacement therapy.

Enzyme therapy in Gaucher disease type 1: dosage efficacy and adverse effects in 33 patients treated for 6 to 24 months.

Principal stratification in causal inference

Understanding the dose-effect relationship: clinical application of pharmacokinetic-pharmacodynamic models.

Prevalence of lysosomal storage disorders.

Enzyme replacement therapy and monitoring for children with type 1 Gaucher disease: consensus recommendations.

Therapeutic response to intravenous infusions of glucocerebrosidase in a patient with Gaucher disease.

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Enzyme replacement therapy

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