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The U.S. Food and Drug Administration (FDA) has cleared a Phase 1/2a trial to test uniQure‘s gene therapy candidate AMT-191 in a small group of people with Fabry disease. UniQure says it expects that enrollment in the U.S.-based trial — which will recruit six patients in all —…
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I walk into the small Jazzercise studio, where I regularly enjoy breaking a sweat, moving to music, and sharing space with upbeat people. Fall decor is on the walls. A rustic sign reads, “It is not happy people who are thankful. It’s thankful people who are happy.” These words ring…
This week, I turned 69 years old. As a man with classic Fabry disease, I consider this achievement both significant and unexpected. I’m still getting used to the idea that living this long is possible for people like me, after believing that it wasn’t for most of my adult…
The first Fabry disease patient has been dosed in a Phase 2 clinical trial that’s testing the experimental oral therapy AL01211. The Phase 2 study (NCT06114329) is expected to enroll 18 male patients with classic Fabry disease, ages 18 to 60, who have not received any prior…
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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