In interviews, people living with Fabry disease in Spain reported a number of unmet needs — among them a general dearth of understanding of the condition’s challenges — that negatively impact patient quality of life. Topping the patients’ main concerns were the scant knowledge about Fabry among work colleagues,…
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In a portion of the population with Fabry disease (FD), strokes and transient ischemic attacks (stroke-like symptoms that last only a short time) are a common manifestation of the disease. As a Fabry patient, the potential for stroke is scary. In 2011, a cousin of mine who had FD…
The European Medicines Agency (EMA) has validated a regulatory application that seeks clearance of a less frequent regimen of Elfabrio (pegunigalsidase alfa), an approved enzyme replacement therapy (ERT) for adults with Fabry disease. If the agency decides to approve the variation application after its review, Fabry patients in…
The U.S. Food and Drug Administration (FDA) has granted orphan drug designation to EXG110, an experimental gene therapy for Fabry disease that’s being developed by Exegenesis Bio and is currently in clinical testing. “The FDA’s decision to grant orphan drug designation to EXG110 highlights the need for better…
Becoming educated and informed about Fabry disease is a good first step. Here you’ll find general information about the disease, including diagnosis, symptoms, and causes.
It helps to know others have been where you are now. Here’s a collection of our columnists’ words of wisdom to help you along on your Fabry disease journey.
There is no cure for Fabry disease yet, but there are treatments that can help manage the disease. Learn more about the approved and experimental therapies here.
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