- Current treatment for Fabry disease involves
enzyme replacement therapy (ERT) with intravenous infusions of recombinant
human α-galactosidase A.
This therapeutic regimen consistently
decreases Gb3 levels in plasma and clears lysosomal inclusions from vascular
endothelial cells.
The effects of ERT on other tissues are not as
obvious, suggesting that treatment must be initiated early in the course of the
disease to be optimally effective or that some complications of the disease are
not responsive to enzymes delivered intravenously.
Enzyme Replacement Therapy
- We use an enzyme called Fabrazyme which is intended to replace the
missing enzyme in
patients with this progressive disease
-Fabrazyme (agalsidase beta) is used to treat patients with Fabry
disease. Fabrazyme lowers the amount of a substance called
globotriaosylceramide (GL-3), which builds up in cells lining the blood vessels
of the kidney and certain other cells.
-The lowering of GL-3 suggests that Fabrazyme may
improve how Fabry disease affects your body; however a relationship of lower
GL-3 to specific signs and symptoms of Fabry disease has not been proven.
Interesting introduction to a less widely known illness!
ReplyDeleteRegarding enzyme replacement therapy via the usage of Fabrazyme, are there any side effects to this treatment? Is it also safe for long-term usage?
hello
ReplyDeleteLife-threatening severe allergic (anaphylactic) reactions have actually been seen in patients during Fabrazyme infusions. The common symptoms are symptoms that are called infusion reactions. Therefore when fabrazyme patients experience a severe allergic or anaphylactic reaction,their healthcare professional will immediately stop the infusion of Fabrazyme and provide them with the necessary emergency medical treatment.Because of the possibility that severe allergic reactions may occur, appropriate medical support should be available during their Fabrazyme infusion.
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