Enzyme replacement therapy in type 1 Gaucher disease and a review of the literature

Gaucher disease (GD) is the most common lysosomal storage disorder. Deficiency of the lysosomal enzyme glucocerebrosidase results in the intracellular accumulation of undegraded substrates in the spleen, liver and bone marrow. Enzyme replacement therapy (ERT) is a standard approach for type 1 GD. He...

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Bibliographic Details
Main Authors: Gökhan Kabaçam, Gülşah Kabaçam, Pervin Topçuoğlu, Işınsu Kuzu, Mutlu Arat
Format: Article
Language:English
Published: Galenos Publishing House 2010-09-01
Series:Turkish Journal of Hematology
Subjects:
Online Access:http://www.journalagent.com/z4/download_fulltext.asp?pdir=tjh&plng=eng&un=TJH-73644