News

Galafold Safe, Effective at Reducing Heart Damage: Real-life Study

Galafold (migalastat) is generally safe and significantly reduces disease-associated heart enlargement in Fabry disease patients carrying Galafold-amenable mutations, according to two-year data from a real-life, multicenter study in Germany. These benefits were generally similar between patients who had previously received standard enzyme replacement therapy (ERT) and those who…

Fabry GLA Gene Variant Found in Parkinson’s Patients

A variant in the GLA gene associated with Fabry disease was found in four women with Parkinson’s disease, but they had no Fabry symptoms, according to results from a study of 236 Parkinson’s patients. According to the research team, the significance of the mutation and the relationship between the two…

Rare Autoimmune Kidney Disorder Seen With Fabry Disease

The case of a man with Fabry disease that co-occurred with a rare autoimmune kidney disorder was described in a recent report. While it’s possible that the rare combination was coincidental, researchers speculated that damage to the kidneys resulting from Fabry disease might have set the stage for the…

Organizations Rally to Help Ukrainian Rare Disease Patients

A Russian military plane crash near Tetiana Zamorska’s home in Kyiv, Ukraine, was a sign that it was time for her and her family to leave. The treacherous, 34-hour pilgrimage that ultimately brought the group of eight by car to temporary accommodations in neighboring Poland last month was physically and emotionally difficult,…

PRX-102 Shows Safety, Efficacy Similar to Fabrazyme in Phase 3 Trial

Two years of treatment with the experimental enzyme replacement therapy PRX-102 (pegunigalsidase alfa) prevented kidney decline in adults with Fabry disease and showed safety and effectiveness similar to Fabrazyme (agalsidase beta), top-line data from the BALANCE Phase 3 clinical trial show. In addition to meeting the trial’s primary goal…

Europe Awaits Proposed New Framework for Sharing Health Data

The European Commission is expected to propose a new governing framework for health data next month, called the European Health Data Space (EHDS), with the aim of connecting national health systems to facilitate secure and efficient transfer of data across systems in different European nations. The move is expected to…