نتایج جستجو برای: chitotriosidase
تعداد نتایج: 246 فیلتر نتایج به سال:
This paper describes the effects of a switch to velaglucerase alfa in a group of adult patients with type 1 Gaucher disease, all of whom had previously had their dose reduced as a consequence of the worldwide imiglucerase shortage. Thirty-two patients from two large European Gaucher centers switched to treatment with velaglucerase alfa after 1-8.5 months of dose reduction. The course of importa...
OBJECTIVES Caspofungin, currently used as salvage therapy for invasive pulmonary aspergillosis (IPA), strangely only causes morphological changes in fungal growth in vitro but does not inhibit the growth. In vivo it has good efficacy. Therefore the question arises how this in vivo activity is reached. Caspofungin is known to increase the amount of chitin in the fungal cell wall. Mammals produce...
The paper by De Fost et al. in the current issue of Blood contains important data with regard to dose-sensitivity in the treatment of Gaucher disease, but the title is misleading. It implies that high-dose enzyme replacement therapy may be superior to low-dose enzyme replacement therapy. However, it is apparent that “superiority” in response is limited to two surrogate markers, serum chitotrios...
Lysosomal integral membrane protein-2 (LIMP2) mediates trafficking of glucocerebrosidase (GBA) to lysosomes. Deficiency of LIMP2 causes action myoclonus-renal failure syndrome (AMRF). LIMP2-deficient fibroblasts virtually lack GBA like the cells of patients with Gaucher disease (GD), a lysosomal storage disorder caused by mutations in the GBA gene. While GD is characterized by the presence of g...
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