Noninvasive echocardiogram imaging may serve as an early marker for left ventricular hypertrophy (LVH), a disease of the left heart muscle, in adults with Fabry disease, a study suggests. Imaging measurements could reliably distinguish Fabry patients among individuals with LVH, with Fabry men, in particular, showing markedly worse…
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In what researchers say is the first reported case of its kind, a woman in China in her 60s was diagnosed with two co-occurring rare conditions: Fabry disease and myelodysplastic syndrome, known as MDS, a disorder in which a person’s bone marrow doesn’t make enough healthy blood cells. The…
Chiesi Global Rare Diseases and Protalix Biotherapeutics are asking a committee of the European Medicines Agency (EMA) to reconsider its recent rejection of a monthly dosing plan for their approved treatment, Elfabrio (pegunigalsidase alfa), for Fabry disease. The proposed regimen would provide patients with a more…
Enzyme replacement therapy (ERT) preserved or improved heart function in two men with Fabry disease who had already experienced kidney failure and were undergoing renal replacement (dialysis or kidney transplant), a new case study reports. The study provides evidence that ERT, the standard treatment for Fabry disease,…
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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