FAQs

Replagal is a treatment for Fabry disease patients ages 7 and older. Shire (now owned by Takeda) developed it. Although in use in many countries, including those of the European Union, the U.S. Food and Drug Administration (FDA) has not approved Replagal. Following are answers to some frequently…

Fabrazyme is an approved treatment for Fabry disease patients age 8 and older. It was developed by Sanofi Genzyme. Following are answers to some frequently asked questions about the treatment. How does Fabrazyme work? Fabrazyme is an enzyme replacement therapy. In patients with Fabry…

Fabry disease is a rare genetic disease characterized by the buildup of a fatty molecule — globotriaosylceramide Gb3 or GL-3 — inside cells, interfering with their function. The accumulation of this molecule can lead to kidney disease, among other problems. The kidneys filter waste…

Fabry disease is a rare disease characterized by the buildup of a fatty molecule (globotriaosylceramide Gb3 or GL-3) inside cells. This accumulation can lead to complications that include kidney disease. Normally, the kidneys filter wastes from the blood. Gb3 buildup can prevent this clearance, allowing waste…

Patients and caregivers of genetic disorders such as Fabry disease often have more questions than answers due to the paucity of easily accessible information. This article answers some frequently asked questions (FAQs) about Fabry disease to provide readers a handy reference. What is Fabry disease? Fabry disease is an…