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1 g-term extension phase in which all received eliglustat.
2 spleen volume and randomized 1:1 to receive eliglustat (50 or 100 mg twice daily; n = 20) or placebo
3 ment would remain so after switching to oral eliglustat, a selective inhibitor of glucosylceramide sy
5 stat), myocardial infarction (one patient on eliglustat), and psychotic disorder (one patient on imig
7 with Gaucher disease type 1, treatment with eliglustat compared with placebo for 9 months resulted i
8 e we report long-term safety and efficacy of eliglustat for 157 patients who received eliglustat in t
10 y 27.77% (95% CI, -32.57% to -22.97%) in the eliglustat group (from 13.89 to 10.17 multiples of norma
13 of eliglustat for 157 patients who received eliglustat in the ENCORE trial; data are available for 4
15 occurred due to palpitations (one patient on eliglustat), myocardial infarction (one patient on eligl
20 ation, 84 (85%) of 99 patients who completed eliglustat treatment and 44 (94%) of 47 patients who com
21 yme Replacement Therapy (ENCORE), at 1 year, eliglustat was noninferior to imiglucerase enzyme therap
23 lts with Gaucher disease type 1 treated with eliglustat who remained in the ENCORE trial for up to 4
24 olute differences between groups all favored eliglustat, with a 1.22-g/dL increase in hemoglobin leve
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