Treatment options for patients with Gaucher disease
Gaucher disease is the most common lysosomal storage disorder due to deficiency of ß-glucocerebrosidase. Since the introduction of Ceredase in 1991, enzyme replacement therapy has been the mainstay of treatment with its major disadvantage of long life dependency on biweekly IV therapy. It was more t...
Main Authors: | , |
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Format: | Article |
Language: | English |
Published: |
SpringerOpen
2016-07-01
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Series: | Egyptian Journal of Medical Human Genetics |
Subjects: | |
Online Access: | http://www.sciencedirect.com/science/article/pii/S1110863016000215 |