Children with Fabry disease typically experience long delays before receiving a diagnosis, despite symptoms affecting multiple organs that often emerge in early childhood, a nationwide study in China has found. Overall, a median diagnostic delay of at least four years was seen among the more than 60 children…
April is Fabry Awareness Month, and advocates around the world are working to uplift and connect the community, increase awareness about Fabry disease, and support fundraising efforts. For this year’s awareness campaign, the Fabry International Network (FIN) has selected the theme: “Living with Fabry, Your Unique Strength.”…
Discussion
I was met with warm and welcoming smiles when I walked into a boutique hotel near Dallas, Texas, for the recent Fabry Support & Information Group (FSIG) Get Together. There, I spotted a familiar face standing next to a display table. It was Nancy Ngotho, the senior patient education…
A monthly dosing regimen for the infusion therapy Elfabrio (pegunigalsidase alfa) has been approved in the European Union for adults with Fabry disease who are stable on enzyme replacement therapy (ERT). Under the newly cleared regimen, approved by the European Commission, eligible patients may now receive the ERT…
Discussion
Every 10 to 11 days, I receive an infusion of one of the two enzyme replacement therapies (ERTs) approved in the U.S. to treat Fabry disease (FD). This is a little more frequent than usual, as most patients receive ERT every two weeks. November 2026 will mark 24 years…
The U.S. Food and Drug Administration (FDA) has granted orphan drug designation to GT-GLA-S03, an experimental cell therapy being developed by Glafabra Therapeutics for Fabry disease. The FDA gives this designation to investigational therapies for diseases that affect fewer than 200,000 people in the U.S. It offers incentives such…
Sangamo Therapeutics said it advanced the rolling submission of a biologics license application (BLA) seeking U.S. accelerated approval of its gene therapy candidate isaralgagene civaparvovec (formerly ST-920) for Fabry disease, following positive data from a clinical trial. Accelerated, or conditional, approval allows experimental therapies to reach the…
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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