The U.S. Food and Drug Administration (FDA) has granted orphan drug designation to GT-GLA-S03, an experimental cell therapy being developed by Glafabra Therapeutics for Fabry disease. The FDA gives this designation to investigational therapies for diseases that affect fewer than 200,000 people in the U.S. It offers incentives such…
Sangamo Therapeutics said it advanced the rolling submission of a biologics license application (BLA) seeking U.S. accelerated approval of its gene therapy candidate isaralgagene civaparvovec (formerly ST-920) for Fabry disease, following positive data from a clinical trial. Accelerated, or conditional, approval allows experimental therapies to reach the…
Routine screening for gastrointestinal (GI) issues should be a standard part of clinical care for Fabry disease, according to a new study that found more than 70% of patients experience GI symptoms. The research highlights that roughly half of adults with the genetic disorder experience moderate to severe digestive…
Discussion
As a columnist for over three years, I have chronicled my family’s journey with Fabry disease by focusing on the experiences of three of my five children who inherited the Fabry mutation from me: twins Michael and Anthony, and Marisa. For this column, I wanted to give a…
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…
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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