Data from an ongoing Phase 1/2 clinical study of ST-920 (isaralgagene civaparvovec), an investigational gene therapy for Fabry…
Articles by Margarida Maia, PhD
An endomyocardial biopsy, a procedure to obtain a small sample of heart muscle, helped doctors reach a definite diagnosis…
The U.S. Food and Drug Administration (FDA) has granted orphan drug status to AMT-191, an investigational one-time gene therapy being…
Early diagnosis and a quick start to enzyme replacement therapy (ERT) with agalsidase alfa eased a patient’s Fabry…
Nearly one-third of Danish patients in Fabry disease registries were found to have poor lung function, with obstructive airflow…
In 2023, Fabry Disease News brought readers consistent coverage of the most recent developments in scientific research and clinical…
A man diagnosed in his 60s with late-onset Fabry disease had an “uneventful” recovery after receiving both a heart and…
Researchers have used stem cells as a “cargo ship” to carry genetic information to produce an enzyme that mimics the…
Measuring the activity of a certain enzyme whose levels are low or absent in Fabry disease may be a…
Idorsia will continue its open-label extension of the Phase 3 MODIFY trial to better determine if lucerastat, the…