Long-term dosing with lucerastat, an experimental substrate reduction treatment from Idorsia, may slow the loss of kidney function in adults with Fabry disease, perhaps by reducing the buildup of toxic fatty molecules in the body’s cells, a global clinical trial has found. The oral therapy — given…
Discussion
The month of January tends to make people bloom with optimism and fresh resolve to achieve their wellness goals. However, the “take control of your health” message doesn’t resonate with people with Fabry disease. This time of year is a reminder of how much is outside of a Fabry…
Markers of disease-related tissue damage in the brains of Fabry disease patients treated with Galafold (migalastat) tend to remain stable over time, a study found. Fabry patients with high blood pressure may be more likely to have damage in the brain, according…
Despite a delayed start until adulthood, enzyme replacement therapy (ERT) was associated with long-term cardiac stability in a man with Fabry disease who had shown symptoms since early childhood, a case study from Brazil shows. Because ERT was initiated before any signs of heart involvement appeared, the man…
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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