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What is Gaucher disease?
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Approved treatments
Enzyme replacement therapy
Cerezyme
Elelyso
VPRIV
Substrate reduction therapy
Cerdelga
Zavesca
Experimental treatments
Chaperone therapy
Ambroxol hydrochloride
Gene therapy
FLT201
PR001
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Splenectomy
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The Gaucher Guide to Grace —
Rivka Silver
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