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  • Title: [Enzyme therapy in children with severe forms of Gaucher's disease].
    Author: Zeman J, Hrebícek M, Vepreková L, Kytnarová J, Houstková H, Malinová V, Hodanová K, Poupĕtová H, Ledvinová J, Hoza J.
    Journal: Cas Lek Cesk; 1997 Feb 05; 136(3):95-7. PubMed ID: 9221177.
    Abstract:
    The enzyme therapy with Ceredase in patients with Gaucher's disease is at present probably the most expensive treatment in the whole world. One-year treatment of an adult patient with Gaucher's disease costs more than 7 million crowns. Indications for treatment in individual patients as well as financial provisions are so far problematic in the Czech Republic. From a total of 28 patients of varying age with Gaucher's disease diagnosed by the authors Ceredase was administered to two boys with a severe course of the disease. Within one year of treatment the health status of both children improved, growth became normal, the spleen diminished in size by 20-35%, haematological manifestations of hypersplenism are receding, there was a 32-46% decline of the activity of serum chitotriosidase and biochemical parameters of the disease improved.
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