Eliglustat maintains long-term clinical stability in patients with Gaucher disease type 1 stabilized on enzyme therapy

Blood

Volumn 129, Issue 17, 2017, Pages 2375-2383

  Cox, Timothy M ^a   Drelichman, Guillermo ^b   Cravo, Renata ^c   Balwani, Manisha ^d   Burrow, Thomas Andrew ^e   Martins, Ana Maria ^f   Lukina, Elena ^g   Rosenbloom, Barry ^h   Goker Alpan, Ozlem ⁱ   Watman, Nora ^j   El Beshlawy, Amal ^k   Kishnani, Priya S ^l   Pedroso, Maria Lucia ^m   Gaemers, Sebastiaan J M ⁿ   Tayag, Regina ^o   Peterschmitt, M Judith ⁿ  

^a UNIVERSITY OF CAMBRIDGE   (United Kingdom)

^b HOSPITAL DE NIÑOS RICARDO GUTIÉRREZ   (Argentina)

^c State Institute of Haematology 'Arthur de Siqueira Cavalcanti'   (Brazil)

^d MOUNT SINAI HOSPITAL   (United States)

^e CINCINNATI CHILDREN'S HOSPITAL MEDICAL CENTER   (United States)

^f FEDERAL UNIVERSITY OF SÃO PAULO   (Brazil)

^g NATIONAL RESEARCH CENTER FOR HEMATOLOGY   (Russian Federation)

^h Cedars Sinai Tower Hematology Oncology   (United States)

ⁱ Dewberry nnd Davis LLC   (United States)

^j HOSPITAL RAMOS MEJÍA   (Argentina)

^k CAIRO UNIVERSITY   (Egypt)

^l DUKE UNIVERSITY SCHOOL OF MEDICINE   (United States)

^m FEDERAL UNIVERSITY OF PARANÁ   (Brazil)

ⁿ GENZYME CORPORATION   (United States)

^o PROMETRIKA LLC   (United States)

more..

Author keywords

[No Author keywords available]

Indexed keywords

ELIGLUSTAT; HEMOGLOBIN; IMIGLUCERASE; ENZYME INHIBITOR; GLUCOSYLCERAMIDASE; PYRROLIDINE DERIVATIVE; RECOMBINANT PROTEIN;

ADULT; ARTHRALGIA; ARTICLE; BONE DENSITY; CONTROLLED STUDY; DIARRHEA; DRUG EFFICACY; DRUG SAFETY; DRUG TOLERABILITY; DRUG WITHDRAWAL; DYSPEPSIA; ENZYME THERAPY; FATIGUE; FEMALE; FEMUR; GASTRITIS; GAUCHER DISEASE; GAUCHER DISEASE TYPE 1; HEART PALPITATION; HEMOGLOBIN DETERMINATION; HUMAN; INTESTINE OBSTRUCTION; LETHARGY; LIVER WEIGHT; LUMBAR SPINE; MAJOR CLINICAL STUDY; MALE; MULTICENTER STUDY; OPEN STUDY; PERIPHERAL NEUROPATHY; PHASE 3 CLINICAL TRIAL; PRIORITY JOURNAL; RANDOMIZED CONTROLLED TRIAL; RASH; SPLEEN SIZE; STATISTICAL ANALYSIS; THROMBOCYTE COUNT; TREATMENT OUTCOME; UNITED STATES; UPPER ABDOMINAL PAIN; VERTIGO; CLINICAL TRIAL; DRUG EFFECTS; ENZYME REPLACEMENT; ENZYMOLOGY; LIVER; LUMBAR VERTEBRA; MIDDLE AGED; ORAL DRUG ADMINISTRATION; PATHOPHYSIOLOGY; SPLEEN;

ADMINISTRATION, ORAL; ADULT; BONE DENSITY; ENZYME INHIBITORS; ENZYME REPLACEMENT THERAPY; FEMALE; FEMUR; GAUCHER DISEASE; GLUCOSYLCERAMIDASE; HUMANS; LIVER; LUMBAR VERTEBRAE; MALE; MIDDLE AGED; PLATELET COUNT; PYRROLIDINES; RECOMBINANT PROTEINS; SPLEEN;

EID: 85018913714     PISSN: 00064971     EISSN: 15280020     Source Type: Journal    
DOI: 10.1182/blood-2016-12-758409     Document Type: Article

References (28)

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