The Comprehensive Gaucher Treatment Center for Gaucher Disease at Tower Hematology Oncology, Beverly Hills
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Gaucher Disease
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Three FDA approved treatments are currently available for the management of Gaucher disease: Cerezyme and VPRIV, both enzyme replacement therapies, as well as Zavesca, a substrate reduction therapy. It is important to note that none of these options is curative, so patients must receive treatment for the rest of their lives in order to maintain the therapeutic benefits.

Cerezyme, developed by Genzyme Corporation, was introduced in 1994 and is considered to be one of the standard treatments for type 1 Gaucher disease. It serves to supplement the body’s naturally occurring enzyme – glucocerebrosidase – that is present in decreased levels among those affected by Gaucher disease. Like glucocerebrosidase, Cerezyme works by breaking down the fatty substance that accumulates in the cells. It is administered intravenously (through the vein), typically on an every 2-week basis. To find out more, visit

VPRIV, developed by Shire Human Genetic Therapies, was approved for commercial use in February 2010 and is also one of the standard treatments for type 1 Gaucher disease. It is a synthetic form of glucocerebrosidase, which utilizes a human cell line technology designed to closely mimic the body’s naturally occurring enzyme. Like Cerezyme, VPRIV is administered intravenously every 2 weeks and functions by breaking down the accumulated substrate from within the cells. For additional information, visit

Zavesca, by Actelion Pharmaceuctials, became available in 2003 and is only indicated for adults with mild-to-moderate type 1 Gaucher disease for whom enzyme replacement therapy is absolutely not an option (i.e. severe allergic reaction). It works by reducing the amount of fatty substance produced by the cells (substrate), thereby enabling the decreased amount of naturally occurring enzyme to better maintain equilibrium. Zavesca is administered orally 3 times a day. To obtain additional information, visit

Research on Gaucher disease has yielded some promising future treatments, including a more targeted substrate inhibitor, a plant-cell based enzyme replacement therapy, gene therapy, stem cell transplantation, and pharmacological chaperoning. For a list of ongoing clinical trials, visit

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