Fatty deposits called myeloid bodies in urine may help diagnose Fabry disease and monitor how people with Fabry are responding to treatment, a study showed. The study, “Urinary Myeloid Bodies as a Biomarker for Early Diagnosis and Monitoring of Enzyme Replacement Therapy in Fabry Disease,” was…
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Picture this: I was soaking in the panorama of emerald mountain peaks framing the blue waters of Lake Amatitlán in Guatemala, where my friend and her groom were kicking off their wedding festivities with friends, food, and music. The event was exceptional, but I was feeling off. An unease had…
Fabry disease patients showed improvements in kidney function in the year following a single dose of gene therapy ST-920 (isaralgagene civaparvovec), and reductions in disease severity were seen at a median of two years after treatment. That’s according to top-line data from the Phase 1/2 STAAR trial…
Since undergoing a heart transplant in September 2020, and after almost 23 years of enzyme replacement therapy to treat my Fabry disease, I have been enjoying relatively good health. My transplanted heart is in great shape. My kidney function has improved significantly. My lung function has returned to a…
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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