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ST-920 (isaralgagene civaparvovec), an experimental gene therapy for Fabry disease that’s currently headed toward Phase 3 clinical testing, has been granted fast track designation by the U.S. Food and Drug Administration (FDA). The FDA gives fast track designation to experimental treatments that have the potential to fill an…
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In Fabry disease, a protein called alpha-synuclein forms clumps inside kidney cells, a toxic buildup that isn’t reversed by conventional treatments. Reducing that buildup can reverse cell damage, opening potential avenues for new treatment strategies, according to “Synuclein [alpha] accumulation mediates podocyte injury in Fabry nephropathy,” which…
More than half of adults with Fabry disease were found to have heart involvement that put them at risk for cardiac events — here, primarily associated with irregular heartbeat and heart failure, according to a new MRI study. While an enlarged heart muscle and scar tissue formation were associated…
PRX-102 (pegunigalsidase alfa) has been approved by the U.S. Food and Drug Administration (FDA) to treat adults with Fabry disease. Now branded Elfabrio (pegunigalsidase alfa-iwxj), the enzyme replacement therapy (ERT) is to be given as an intravenous, or into the vein, infusion every two weeks. It was codeveloped…
Columnist Jerry Walter describes a family camp weekend in North Carolina designed for the Fabry disease community.
Check out Fabry Disease News’ collection of health insights, where you can find a variety of information for patients and caregivers.
Find more answers to your questions about Fabry disease in our FAQs section.
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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