Modelling Gaucher disease progression: Long-term enzyme replacement therapy reduces the incidence of splenectomy and bone complications

Orphanet Journal of Rare Diseases

Volumn 9, Issue 1, 2014, Pages

  Van Dussen, Laura ^a   Biegstraaten, Marieke ^a   Dijkgraaf, Marcel G W ^a   Hollak, Carla E M ^a  

^a ACADEMIC MEDICAL CENTER   (Netherlands)

Author keywords

[No Author keywords available]

Indexed keywords

IMIGLUCERASE; RECOMBINANT ENZYME; TALIGLUCERASE ALFA; VELAGLUCERASE ALFA;

AMYLOIDOSIS; ARTICLE; BONE COMPLICATION; BONE DISEASE; CONTROLLED STUDY; CYTOPENIA; DISEASE COURSE; ENZYME REPLACEMENT; FEMALE; FOLLOW UP; GAUCHER DISEASE; HISTORY; HUMAN; INCIDENCE; LIVER CELL CARCINOMA; LONG TERM CARE; MAJOR CLINICAL STUDY; MALE; MULTIPLE MYELOMA; NETHERLANDS; PARKINSON DISEASE; PULMONARY HYPERTENSION; RETROSPECTIVE STUDY; SPLENECTOMY; SURVIVAL RATE; TIME; ADULT; COMPLICATION; PATHOPHYSIOLOGY;

ADULT; BONE DISEASES; DISEASE PROGRESSION; ENZYME REPLACEMENT THERAPY; GAUCHER DISEASE; HUMANS; NETHERLANDS; SPLENECTOMY;

EID: 84906929955     PISSN: None     EISSN: 17501172     Source Type: Journal    
DOI: 10.1186/s13023-014-0112-x     Document Type: Article

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