• Terapevt Arkh · Jul 2019

    [Enzyme replacement therapy in adult patients with type I Gaucher disease].

    • R V Ponomarev and E A Lukina.
    • National Research Center for Hematology.
    • Terapevt Arkh. 2019 Jul 15; 91 (7): 127-131.

    AbstractEnzyme replacement therapy (ERT) is the standard for the treatment of Gaucher disease (GD). A lifelong intravenous administration of a recombinant analogue of human glucocerebrosidase compensates for the functional deficiency of its own enzyme. The use of ERT has changed the clinical phenotype of GD, a severe progressive disease has been turned into the status of an asymptomatic metabolic defect. At the same time, a reduced dosing ERT regimen applied in Gaucher patients who had achieved therapeutic goals has not yet been developed.

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