Biochemical response to substrate reduction therapy versus enzyme replacement therapy in Gaucher disease type 1 patients

Orphanet Journal of Rare Diseases

Volumn 11, Issue 1, 2016, Pages

  Smid, Bouwien E ^a   Ferraz, Maria J ^a   Verhoek, Marri ^b   Mirzaian, Mina ^a   Wisse, Patrick ^b   Overkleeft, Herman S ^b   Hollak, Carla E ^a   Aerts, Johannes M ^b,c  

^a ACADEMIC MEDICAL CENTER   (Netherlands)

^b LEIDEN UNIVERSITY   (Netherlands)

^c LEIDEN UNIVERSITY   (Netherlands)

Author keywords

Chitotriosidase; Eliglustat; Enzyme replacement therapy; Gaucher disease; Glucosylceramide; Glucosylsphingosine; Miglustat

Indexed keywords

CHEMOKINE; CHEMOKINE CCL18; CHITOTRIOSIDASE; ELIGLUSTAT; GLUCOSYLCERAMIDE; GLUCOSYLSPHINGOSINE; GLYCOSPHINGOLIPID; IMIGLUCERASE; MIGLUSTAT; UNCLASSIFIED DRUG; VELAGLUCERASE ALFA; 1 DEOXYNOJIRIMYCIN; ENZYME INHIBITOR; GLUCOSYLCERAMIDASE; PYRROLIDINE DERIVATIVE;

ADIPOSE TISSUE; ADULT; AGED; ANEMIA; ARTICLE; BONE DISEASE; BONE MARROW FAT FRACTION; CHILD; CLINICAL ARTICLE; COMPARATIVE STUDY; CONTROLLED STUDY; DISEASE SEVERITY; DRUG RESPONSE; DRUG SUBSTITUTION; DRUG THERAPY; DRUG TREATMENT FAILURE; DRUG WITHDRAWAL; ENZYME REPLACEMENT; ENZYME SUBSTRATE; EXPLORATORY RESEARCH; FEMALE; GAUCHER DISEASE; HEMOGLOBIN BLOOD LEVEL; HUMAN; LIPID BLOOD LEVEL; LIVER WEIGHT; MALE; MIDDLE AGED; PROTEIN BLOOD LEVEL; RETROSPECTIVE STUDY; SCHOOL CHILD; SPLEEN WEIGHT; SPLENECTOMY; SUBSTRATE REDUCTION THERAPY; THROMBOCYTE COUNT; THROMBOCYTOPENIA; UNSPECIFIED SIDE EFFECT; ANALOGS AND DERIVATIVES; ANTAGONISTS AND INHIBITORS; ENZYMOLOGY; METABOLISM; PROCEDURES;

1-DEOXYNOJIRIMYCIN; ENZYME INHIBITORS; ENZYME REPLACEMENT THERAPY; FEMALE; GAUCHER DISEASE; GLUCOSYLCERAMIDASE; GLUCOSYLCERAMIDES; HUMANS; MALE; PYRROLIDINES;

EID: 84962427380     PISSN: None     EISSN: 17501172     Source Type: Journal    
DOI: 10.1186/s13023-016-0413-3     Document Type: Article

References (53)

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