Force majeure: therapeutic measures in response to restricted supply of imiglucerase (Cerezyme) for patients with Gaucher disease. - Wikidata - awgldk - TriplyDB

Force majeure: therapeutic measures in response to restricted supply of imiglucerase (Cerezyme) for patients with Gaucher disease.

Force majeure: therapeutic measures in response to restricted supply of imiglucerase (Cerezyme) for patients with Gaucher disease.

http://www.wikidata.org/entity/Q39072981

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Enzyme replacement and substrate reduction therapy for Gaucher disease Enzyme replacement and substrate reduction therapy for Gaucher disease Imiglucerase in the treatment of Gaucher disease: a history and perspective Putting the Spotlight Back on Plant Suspension Cultures The French Gaucher's disease registry: clinical characteristics, complications and treatment of 562 patients.Velaglucerase alfa enzyme replacement therapy compared with imiglucerase in patients with Gaucher disease.Recurrent pulmonary aspergillosis and mycobacterial infection in an unsplenectomized patient with type 1 Gaucher disease Outcome of pregnancies in women receiving velaglucerase alfa for Gaucher disease.The European Gaucher Alliance: a survey of member patient organisations' activities, healthcare environments and concerns.Gaucher disease and its treatment options.Gaucher disease: clinical profile and therapeutic developments.Impact of imiglucerase supply shortage on clinical and laboratory parameters in Norrbottnian patients with Gaucher disease type 3 Limitations of drug registries to evaluate orphan medicinal products for the treatment of lysosomal storage disorders.Direct site-specific glycoform identification and quantitative comparison of glycoprotein therapeutics: imiglucerase and velaglucerase alfa A phase 2 multi-center, open-label, switch-over trial to evaluate the safety and efficacy of Abcertin® in patients with type 1 Gaucher disease.Impact of imiglucerase supply constraint on the therapeutic management and course of disease in French patients with Gaucher disease type 1 Substrate reduction therapy with miglustat for type 1 Gaucher disease: a retrospective analysis from a single institution.Switching from imiglucerase to miglustat for the treatment of French patients with Gaucher disease type 1: a case series.Compassionate use of orphan drugs.An evidence-based review of the potential benefits of taliglucerase alfa in the treatment of patients with Gaucher disease Investigations on therapeutic glucocerebrosidases through paired detection with fluorescent activity-based probes.Evaluation of miglustat as maintenance therapy after enzyme therapy in adults with stable type 1 Gaucher disease: a prospective, open-label non-inferiority study Did the temporary shortage in supply of imiglucerase have clinical consequences? Retrospective observational study on 34 italian Gaucher type I patients.Safety and efficacy of velaglucerase alfa in Gaucher disease type 1 patients previously treated with imiglucerase.Effects of switching from a reduced dose imiglucerase to velaglucerase in type 1 Gaucher disease: clinical and biochemical outcomes 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 Treatment Enzyme replacement therapy with taliglucerase alfa: 36-month safety and efficacy results in adult patients with Gaucher disease previously treated with imiglucerase.Recombinant plant-derived pharmaceutical proteins: current technical and economic bottlenecks.Emerging therapeutic targets for Gaucher disease.Generation of a Chinese hamster ovary cell line producing recombinant human glucocerebrosidase.Enrichment of apoplastic fluid with therapeutic recombinant protein for efficient biofarming.Endosperm-specific expression of human acid beta-glucosidase in a waxy rice Taliglucerase alfa: safety and efficacy across 6 clinical studies in adults and children with Gaucher disease.Pharmacotherapy of Gaucher Disease: Current and Future Options.Contained Molecular Farming Using Plant Cell and Tissue Cultures

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Force majeure: therapeutic measures in response to restricted supply of imiglucerase (Cerezyme) for patients with Gaucher disease.

http://www.wikidata.org/entity/Q39072981

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J.BCMD.2009.09.006

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Blood Cells, Molecules and Diseases

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Force majeure: therapeutic mea ...... patients with Gaucher disease.

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Ari Zimran

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Bruno Bembi

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Carla E M Hollak

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Maria Clara Sa Miranda

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scholarly article

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10.1016/J.BCMD.2009.09.006

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Anna Tylki-Szymańska

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