Fabry Disease - Treatment

Treatment

Until the 2000s, treatment of Fabry disease targeted the symptomatic effects.

In 2001, two Enzyme Replacement Therapies (ERTs) were released which attempt to replace the deficient enzyme by means of infusion. The drugs are expensive, with an annual cost of approximately $200,000 US per patient, and that constitutes a barrier to successful treatment for many patients around the world. Enzyme replacement therapy is not a cure, but can allow improved metabolism and partially prevent disease progression, as well as potentially reversing some symptoms.

Pain associated with Fabry disease can be partially alleviated by ERT, but pain management regimens may also include analgesics, anticonvulsants, and non-steroidal anti-inflammatory drugs.

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