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Budget Impact Analysis of Eliglustat for the Treatment of Gaucher Disease Type 1 in the United States.

著者 Nalysnyk L , Sugarman R , Cele C , Uyei J , Ward A
J Manag Care Spec Pharm.2018 Oct ; 24(10):1002-1008.
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Gaucher disease type 1 (GD1) is a rare, genetic, lysosomal storage disease with no cure. Current treatment options include intravenous (IV) enzyme replacement therapy ([ERT]; imiglucerase, velaglucerase alfa, or taliglucerase alfa) or oral substrate reduction therapy ([SRT]; eliglustat or miglustat). The cost to U.S. payers of an IV-administered drug can vary depending on the site of care (i.e., home, outpatient clinic, or hospital setting). Treatment with oral eliglustat may present an opportunity for cost savings.
PMID: 30247105 [PubMed - in process]
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