Enzyme Replacement Therapy (ERT) is a medical treatment replacing an enzyme in patients in whom that particular enzyme is deficient or absent. In the case of Fabry disease, the enzyme that is deficient is called α‐galactosidase A (α‐Gal A) enzyme. ERT was approved in Canada for the treatment of Fabry disease in 2004 and should be considered in all patients with documented Fabry disease, of any age and either sex, who meet at least one of the disease-specific criteria.
The most recent clinical guidelines and criteria for the use of ERT in Canada can be read in detail here.
Fabrazyme (agalsidase beta) is used as ERT for people with Fabry disease, which is a genetic disorder where the levels of α‐galactosidase A (α‐Gal A), an enzyme that breaks down fats, is low or absent. With Fabry disease, globotriaosylceramide (or GL-3; a fat substance) is not removed from cells in the body and starts to accumulate in the walls of blood vessels and organs. By reducing GL-3 levels in the body, Fabrazyme slows the rate of progression of Fabry disease in the kidney, heart, and brain.
To view the Fabrazyme Product Monograph, please click here.
The Fabry Registry, sponsored by Sanofi Genzyme, has been established in order to better understand the variability and progression of Fabry disease, and to continue to monitor and evaluate safety and effectiveness of Fabrazyme®. You are encouraged to participate.
Information regarding the registry program may be found at RegistryNXT.com.
If you are interested in participating, please contact your doctor.
For more information, please visit SanofiGenzyme.ca.
Research is ongoing and new therapies are currently under development.
Please visit our News & Events section for the latest updates in Fabry disease research.
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