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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
4  we randomly allocated 106 (66%) patients to eliglustat and 54 (34%) to imiglucerase.
5 stat), myocardial infarction (one patient on eliglustat), and psychotic disorder (one patient on imig
6 y lived in the United States when commercial eliglustat became available.
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
9 a are available for 46 patients who received eliglustat for 4 years.
10 y 27.77% (95% CI, -32.57% to -22.97%) in the eliglustat group (from 13.89 to 10.17 multiples of norma
11              97 (92%) of 106 patients in the eliglustat group had treatment-emergent adverse events,
12                           One patient in the eliglustat group withdrew (non-treatment related); 39 of
13  of eliglustat for 157 patients who received eliglustat in the ENCORE trial; data are available for 4
14                                         Oral eliglustat maintained haematological and organ volume st
15 occurred due to palpitations (one patient on eliglustat), myocardial infarction (one patient on eligl
16 -generated centrally) to receive either oral eliglustat or imiglucerase infusions for 12 months.
17                                  Duration on eliglustat ranged from 2 to 5 years, depending on timing
18       The non-inferiority margin was 25% for eliglustat relative to imiglucerase, assessed in all pat
19                      In the phase 3 Study of Eliglustat Tartrate (Genz-112638) in Patients With Gauch
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
22                                              Eliglustat was well tolerated over 4 years; 4 (2.5%) pat
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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