Clinical response to eliglustat in treatment-naïve patients with Gaucher disease type 1: Post-hoc comparison to imiglucerase-treated patients enrolled in the International Collaborative Gaucher Group Gaucher Registry

Molecular Genetics and Metabolism Reports

Volumn 8, Issue , 2016, Pages 17-19

  Ibrahim, Jennifer ^a   Underhill, Lisa H ^a   Taylor, John S ^a,b   Angell, Jennifer ^a   Peterschmitt, M Judith ^a  

^a GENZYME CORPORATION   (United States)

^b JOHNSON AND JOHNSON   (United States)

Author keywords

Acid glucosidase deficiency; Eliglustat; Enzyme replacement therapy; Gaucher disease type 1; Imiglucerase; Substrate reduction therapy

Indexed keywords

ELIGLUSTAT; HEMOGLOBIN; IMIGLUCERASE; PLACEBO;

ADULT; ARTICLE; CONTROLLED STUDY; DISEASE REGISTRY; FEMALE; GAUCHER DISEASE; HEMOGLOBIN BLOOD LEVEL; HUMAN; LIVER WEIGHT; MAJOR CLINICAL STUDY; MALE; ORGAN SIZE; PHASE 2 CLINICAL TRIAL (TOPIC); PHASE 3 CLINICAL TRIAL (TOPIC); POST HOC ANALYSIS; SPLEEN; SPLEEN VOLUME; THROMBOCYTE COUNT; TREATMENT RESPONSE;

EID: 84976338409     PISSN: None     EISSN: 22144269     Source Type: Journal    
DOI: 10.1016/j.ymgmr.2016.06.003     Document Type: Article

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