The effective management of Fabry disease requires a multidisciplinary approach. Comprehensive therapy includes intravenously administered enzyme replacement therapy (ERT), conventional medical treatment, and adjunct therapies, and may include lifestyle modifications and prophylactic medications.1

In the United States and many other countries there is a commercially approved Enzyme Replacement Therapy (ERT) to treat Fabry disease called agalsidase beta (Fabrazyme®) produced by Sanofi-Genzyme. It is administered by intravenous infusion usually every two weeks for life or until another suitable medication becomes available. The approved dose is 1 milligrams per kilogram of body weight.

In Europe and many other countries but not in the United States, there is a commercially approved intravenous Enzyme Replacement Therapy (ERT) to treat Fabry disease called agalsidase alfa (Replagal®) produced by Shire. It is also administered by intravenous infusion usually every two weeks for life or until another suitable medication becomes available. The approved dose is 0.2 milligrams per kilogram of body weight.

Medications such as antihistamines and others are often given prior to infusions to lessen the chance of infusion related reactions and sometimes are given after infusions. Some medications like amiordarone and qloroquine may interfere with the effectiveness of Fabrazyme(R). Consult your physician.

Proteinuria, if present, is often treated along with other therapies to prolong kidney health.

Several other treatments are being investigated in clinical trials or are planned to begin investigated in clinical trials. Other treatments being investigated include:  an oral chaperone therapy (Amicus), a plant-based ERT (Protalix), a substrate reduction therapy (Sanofi-Genzyme), a potentially bio-better ERT (Codexis), a gene editing solution (Sangamo), and a gene therapy solution (Canadian Institutes of Health, et al.)

Reference: 1. Germain DP: Fabry disease. Orphanet J Rare Dis 2010, 5:30.

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