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Is Fabry Disease Treatment Available?

Fabry disease can be treated! 
 
In the  U.S., one Enzyme Replacement Therapy (ERT), Fabrazyme®, was approved by the Food and Drug Administration (FDA) in April, 2003. The therapy was conditionally approved in an accelerated approval process that involves further study of the new treatment to confirm clinical benefit.  In many other countries, two ERT treatment medications, Replagal® and Fabrazyme®, are approved.     
 
The most current medical study in September 2006 (link below), indicates:
 
"... Based on current knowledge, every male Fabry patient should be offered ERT, irrespective of the of the stage of CKD [chronic kidney disease] ...." and  

"... Females should be offered ERT if they manifest significant symptoms or show evidence of progressive end organ Fabry involvement including chronic acroparesthesias resistant to conventional therapy, persistent proteinuria (> 300 mg/24 hours), GFR below 80 mL/minute/1.73 m2, clinically significant cardiac involvement, a previous cerebrovascular accident or history of transient ischemic attacks, or ischemic changes on brain MRI ....  Other possible conditions include symptoms unresponsive to other forms of medical management, including chronic, disabling gastro-intestinal dysfunction, pulmonary involvement, or abnormal blood pressure regulation during exercise testing...."  
 
Fabry disease: Guidelines for the evaluation and of multi-organ system involvement

Note: While there are proven significant clinical benefit to receiving enzyme replacement therapy (ERT), treatment may not remedy irreversible organ and system damage. Even with clinical benefit to the heart, kidneys, and neurological system, treatment may not significantly remedy quality of life symptoms. While many individuals feel markedly better with treatment, many individulas on ERT report varying degrees of continued pain, edema, inability to sweat, low energy, diarrhea, vertigo, and other symptoms.


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