Children with Fabry disease typically experience long delays before receiving a diagnosis, despite symptoms affecting multiple organs that often emerge in early childhood, a nationwide study in China has found. Overall, a median diagnostic delay of at least four years was seen among the more than 60 children…
News
April is Fabry Awareness Month, and advocates around the world are working to uplift and connect the community, increase awareness about Fabry disease, and support fundraising efforts. For this year’s awareness campaign, the Fabry International Network (FIN) has selected the theme: “Living with Fabry, Your Unique Strength.”…
A monthly dosing regimen for the infusion therapy Elfabrio (pegunigalsidase alfa) has been approved in the European Union for adults with Fabry disease who are stable on enzyme replacement therapy (ERT). Under the newly cleared regimen, approved by the European Commission, eligible patients may now receive the ERT…
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…
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…
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…
Recent Posts
- Despite disease symptoms, long delays seen for a Fabry diagnosis in children
- Fabry Awareness Month focuses on community, strength, and support
- Finding my tribe at FSIG’s recent Fabry community get-together
- New monthly dosing of Elfabrio approved in EU for some Fabry patients
- Understanding the significance of lyso-Gb3 in Fabry disease
- FDA grants orphan designation to new Fabry cell therapy GT-GLA-S03
- Sangamo seeks accelerated US approval of gene therapy for Fabry
- Common Fabry symptoms often mimic IBS in adults
- Two of my sons share what it’s like having three siblings with Fabry
- Idorsia outlines new Phase 3 program for lucerastat in Fabry disease