Fabry Disease is a genetic disorder in which there is an inadequate level of an enzyme called α-galactosidase produced by the body. Without adequate a-galactosidase, a substance called “Gb3 or GL-3” accumulates in vulnerable cells and eventually causes damage. Symptoms include pain in the extremities, heat intolerance, stomach aches, inability to sweat and skin lesions such as angiokeratomas. Over time vital organs such as the kidneys, heart and blood vessels in the brain can be affected.
Replagal® (agalsidase alfa ghu)
Replagal is a human a-galactosidase A produced by genetic engineering technology. Replagal is an enzyme replacement therapy (ERT) which helps to break down Gb3 (GL-3). Replagal is given by intravenous infusion over 40minutes every two weeks.
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