ライフサイエンスコーパス: eliglustat

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 Eliglustat, a U.S. Food and Drug Administration-approved

5 we randomly allocated 106 (66%) patients to eliglustat and 54 (34%) to imiglucerase.

6 stat), myocardial infarction (one patient on eliglustat), and psychotic disorder (one patient on imig

7 osteoclast differentiation, was inhibited by eliglustat as evidenced by TRAF3 lysosomal and cytoplasm

8 y lived in the United States when commercial eliglustat became available.

9 Eliglustat blocked autophagy by altering glycosphingolip

10 with Gaucher disease type 1, treatment with eliglustat compared with placebo for 9 months resulted i

11 e we report long-term safety and efficacy of eliglustat for 157 patients who received eliglustat in t

12 a are available for 46 patients who received eliglustat for 4 years.

13 ting the clinical translational potential of eliglustat for the treatment of myeloma bone disease.

14 y 27.77% (95% CI, -32.57% to -22.97%) in the eliglustat group (from 13.89 to 10.17 multiples of norma

15 97 (92%) of 106 patients in the eliglustat group had treatment-emergent adverse events,

16 One patient in the eliglustat group withdrew (non-treatment related); 39 of

17 of eliglustat for 157 patients who received eliglustat in the ENCORE trial; data are available for 4

18 Oral eliglustat maintained haematological and organ volume st

19 occurred due to palpitations (one patient on eliglustat), myocardial infarction (one patient on eligl

20 -generated centrally) to receive either oral eliglustat or imiglucerase infusions for 12 months.

21 nhibition of glucosylceramide synthesis with eliglustat, partially corrected the impaired lactosylcer

22 hosphonate that treats myeloma bone disease, eliglustat provided further protection from bone loss.

23 Duration on eliglustat ranged from 2 to 5 years, depending on timing

24 The non-inferiority margin was 25% for eliglustat relative to imiglucerase, assessed in all pat

25 No patient receiving eliglustat SRT suffered AVN.

26 rminants of AVN in patients receiving ERT or eliglustat substrate reduction therapy (SRT) during 20 y

27 In the phase 3 Study of Eliglustat Tartrate (Genz-112638) in Patients With Gauch

28 ation, 84 (85%) of 99 patients who completed eliglustat treatment and 44 (94%) of 47 patients who com

29 yme Replacement Therapy (ENCORE), at 1 year, eliglustat was noninferior to imiglucerase enzyme therap

30 Eliglustat was well tolerated over 4 years; 4 (2.5%) pat

31 lts with Gaucher disease type 1 treated with eliglustat who remained in the ENCORE trial for up to 4

32 olute differences between groups all favored eliglustat, with a 1.22-g/dL increase in hemoglobin leve