Standard newborn screening tests for Fabry disease regularly miss female infants, leading to delays…
Articles by Marisa Wexler, MS
A new guidebook aims to support young adults living with Fabry disease. The Fabry International Network (FIN) developed…
The U.S. Food and Drug Administration (FDA) has agreed to meet with Glafabra Therapeutics to discuss plans for clinical…
A team of scientists in Spain has developed a new experimental nanoparticle-based treatment approach that aims to address the root…
April is Fabry Awareness Month, and advocates around the world are working to uplift and connect the community, increase awareness…
The U.S. Food and Drug Administration (FDA) has granted orphan drug designation to GT-GLA-S03, an experimental cell therapy being developed…
Following discussions with regulatory authorities in the U.S. and Europe, Idorsia has outlined a new Phase 3 registration program…
People with Fabry disease have abnormalities in the blood vessels within the eye, and these abnormalities may be indicative…
The experimental gene therapy AMT-191 appears to be working as intended in an early clinical trial, developer uniQure said,…
Sangamo Therapeutics has begun seeking accelerated approval from the U.S. Food and Drug Administration (FDA) for isaralgagene…