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enzyme replacement therapy (7) 7
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imiglucerase (6) 6
eliglustat (5) 5
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Journal Article
Blood Cells, Molecules and Diseases, ISSN 1079-9796, 02/2018, Volume 68, pp. 185 - 191
Eliglustat, an oral substrate reduction therapy, is a first-line therapy for adults with Gaucher disease type 1 and a compatible CYP2D6 metabolizer phenotype.... 
Acid β-glucosidase deficiency | Gaucher disease type 1 | Eliglustat | Safety | Adverse events | Substrate reduction therapy | DIAGNOSIS | REDUCTION | TARTRATE | PHENOTYPE | HEMATOLOGY | Acid beta-glucosidase deficiency | ENZYME REPLACEMENT THERAPY | Diarrhea | Clinical trials | Complications and side effects | Adults | Backache | Cytochrome P-450
Journal Article
American Journal of Hematology, ISSN 0361-8609, 01/2019, Volume 94, Issue 1, pp. 29 - 38
Eliglustat is a first‐line oral therapy for adults with Gaucher disease type 1 (GD1) and poor, intermediate or extensive CYP2D6‐metabolizer phenotypes (>90% of... 
GLUCOSYLSPHINGOSINE | DOUBLE-BLIND | ADULTS | SEVERITY SCORING SYSTEM | ENGAGE | SCALE | HEMATOLOGY | ENZYME REPLACEMENT THERAPY | GOALS | IMIGLUCERASE
Journal Article
Molecular Genetics and Metabolism Reports, ISSN 2214-4269, 12/2016, Volume 9, pp. 25 - 28
Gaucher disease type 1 is an autosomal recessive disorder caused by deficient activity of the lysosomal enzyme acid β-glucosidase resulting in accumulation of... 
Imiglucerase | Gaucher disease type 1 | Eliglustat | Velaglucerase alfa | Composite endpoint | Non-inferiority trial | EFFICACY | TARTRATE | SAFETY | OPEN-LABEL | PHASE-2 | NON-INFERIORITY | THERAPY | GENETICS & HEREDITY | ORAL ELIGLUSTAT
Journal Article
Blood, ISSN 0006-4971, 12/2015, Volume 126, Issue 23, pp. 884 - 884
Introduction: Gaucher disease is caused by a deficiency in lysosomal acid β-glucosidase, which leads to accumulation of glucosylceramide primarily in... 
Journal Article
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