Approved Treatments

Enzyme Replacement Therapy

Enzyme replacement therapy deals with the underlying cause of Fabry disease — mutations of the GLA gene — by generating the enzyme that is non-functional or missing in a disease. It is a lifelong treatment, with the enzyme given continuously.

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Chaperone Therapy

Chaperones are small molecules that assist proteins in becoming functional by helping them take the correct shape and stay stable. With chaperone therapy, some faulty forms of alpha-galactosidase A can be corrected and delivered to the lysosomes so that the excess globotriaosylceramide can be broken down.

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Other Treatment Strategies

Accumulation of the fat molecule globotriaosylceramide causes several characteristic symptoms of Fabry disease, including kidney disease. If not treated, kidney disease can cause potentially life-threatening kidney failure. Dialysis can help supplement kidney function; in severe cases, a kidney transplant may be necessary.