Having Fabry disease means living in an unpredictable body and in a world full of people who often refuse to believe what they can’t see. I have good days when movement feels almost normal, pain sits quietly in the background, and energy allows for work, laughter, and making plans.
Sangamo Therapeutics has begun seeking accelerated approval from the U.S. Food and Drug Administration (FDA) for isaralgagene civaparvovec (formerly ST-920), its experimental gene therapy for Fabry disease. Accelerated approval is a type of conditional approval in which the FDA allows a therapy to be sold based…
Discussion
Since writing my last column in December, I’ve been working to find the most effective exercise routine and remedies to prevent my blood pressure (BP) from rising too high. Uncontrolled hypertension can lead to heart attack, stroke, and other serious health issues. Until recently, like many people with…
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…
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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