Eliglustat, an investigational oral therapy for gaucher disease type 1: Phase 2 trial results after 4years of treatment

Blood Cells, Molecules, and Diseases

Volumn 53, Issue 4, 2014, Pages 274-276

  Lukina, Elena ^a   Watman, Nora ^b   Dragosky, Marta ^c   Pastores, Gregory M ^d   Arreguin, Elsa Avila ^e   Rosenbaum, Hanna ^f   Zimran, Ari ^g   Angell, Jennifer ^h   Ross, Leorah ^h   Puga, Ana Cristina ^h   Peterschmitt, Judith M ^h  

^a N N BLOKHIN NATIONAL MEDICAL RESEARCH CENTER OF ONCOLOGY   (Russian Federation)

^b HOSPITAL RAMOS MEJÍA   (Argentina)

^c Instituto Argentino de Diagnostico y Tratamiento   (Argentina)

^d National Gaucher Disease Treatment Center: Yale Lysosomal Disease Center   (United States)

^e HOSPITAL DE ESPECIALIDADES   (Mexico)

^f RAMBAM MEDICAL CENTER   (Israel)

^g SHAARE ZEDEK MEDICAL CENTER   (Israel)

^h GENZYME CORPORATION   (United States)

Author keywords

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

Indexed keywords

BETA GLUCOSIDASE; CCL18 CHEMOKINE; CHEMOKINE; CHITOTRIOSIDASE; ELIGLUSTAT; GANGLIOSIDE GM3; GLUCOSYLCERAMIDE; HEMOGLOBIN; UNCLASSIFIED DRUG; BETA CHEMOKINE; BETA N ACETYLHEXOSAMINIDASE; CCL18 PROTEIN, HUMAN; ENZYME INHIBITOR; NEW DRUG; PYRROLIDINE DERIVATIVE;

ADULT; ARTICLE; BONE DENSITY; CLINICAL ARTICLE; CONTROLLED STUDY; DRUG ADMINISTRATION ROUTE; DRUG EFFICACY; DRUG SAFETY; ENZYME BLOOD LEVEL; FEMALE; FEMUR; GAUCHER DISEASE; HEMOGLOBIN BLOOD LEVEL; HUMAN; LIVER SIZE; LONG TERM CARE; LUMBAR SPINE; MALE; MULTICENTER STUDY; OUTCOME ASSESSMENT; PHASE 2 CLINICAL TRIAL; SINGLE BLIND PROCEDURE; SPLEEN SIZE; THROMBOCYTE COUNT; TREATMENT DURATION; UNSPECIFIED SIDE EFFECT; UPREGULATION; ADOLESCENT; BLOOD; BONE MARROW; CLINICAL TRIAL; DRUG EFFECTS; FOLLOW UP; LUMBAR VERTEBRA; METABOLISM; MIDDLE AGED; PATHOLOGY;

ADOLESCENT; ADULT; BONE DENSITY; BONE MARROW; CHEMOKINES, CC; DRUGS, INVESTIGATIONAL; ENZYME INHIBITORS; FEMALE; FOLLOW-UP STUDIES; G(M3) GANGLIOSIDE; GAUCHER DISEASE; GLUCOSYLCERAMIDES; HEMOGLOBINS; HEXOSAMINIDASES; HUMANS; LUMBAR VERTEBRAE; MALE; MIDDLE AGED; PLATELET COUNT; PYRROLIDINES;

EID: 84908086749     PISSN: 10799796     EISSN: 10960961     Source Type: Journal    
DOI: 10.1016/j.bcmd.2014.04.002     Document Type: Article

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