Following discussions with regulatory authorities in the U.S. and Europe, Idorsia has outlined a new Phase 3 registration program to evaluate its experimental oral therapy, lucerastat, for its effects on kidney outcomes in people with Fabry disease. If the studies are successful, Idorsia…
People with Fabry disease have abnormalities in the blood vessels within the eye, and these abnormalities may be indicative of heart disease in Fabry patients, a first-of-its-kind study reports. Researchers also found an association between eye vessel abnormalities and more inflammation in people with certain Fabry-causing mutations…
Discussion
Recently, the Fabry disease (FD) community received disappointing news about an investigational oral treatment called venglustat. Results from the PERIDOT Phase 3 trial, which was evaluating venglustat in FD patients, did not meet its primary endpoint of reducing neuropathic and abdominal pain. Neuropathic pain is one of the…
The experimental gene therapy AMT-191 appears to be working as intended in an early clinical trial, developer uniQure said, but the company is pausing part of the study pending further investigation into severe liver damage cases. “While the study remains ongoing, we believe the preliminary data collected are supportive…
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…
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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