Fabry disease patients showed improvements in kidney function in the year following a single dose of gene therapy ST-920 (isaralgagene civaparvovec), and reductions in disease severity were seen at a median of two years after treatment. That’s according to top-line data from the Phase 1/2 STAAR trial…
Levels of certain molecules in the blood may help track disease activity and kidney damage in Fabry disease, a study found. “These biomarkers may play a crucial role in improving clinical management by facilitating early diagnosis and personalized treatment strategies for” Fabry disease, researchers wrote in the study, “…
For Fabry Awareness Month this April, the Fabry International Network, or FIN — an alliance of 61 patient organizations in 57 countries — is shining a spotlight on doctors who have focused on caring for people with the rare inherited disease, helping patients better understand the disorder, get accurate…
People with different lysosomal storage diseases have varying beliefs about prescribed medicines, which reflect their current medical needs, a study has found. Particularly, attitudes toward treatment in Fabry disease vary according to gender and are different in Fabry and other related disorders. According to researchers, these beliefs should be…
A Fabry disease diagnosis for a woman in Portugal came only after she had a stroke in her early 40s, according to a new report in which the scientists highlighted “the importance of early diagnosis,” especially in nontypical cases. “The observed cerebrovascular [brain blood flow] complications [in this case]…
Throughout 2024, the team at Fabry Disease News brought our readers all the latest updates on research related to Fabry disease and advances in diagnosis and treatment. Here is a list of the 10 most-read articles of last year. We look forward to continuing to be a resource to…
The U.S. Food and Drug Administration (FDA) has granted orphan drug designation to EXG110, an experimental gene therapy for Fabry disease that’s being developed by Exegenesis Bio and is currently in clinical testing. “The FDA’s decision to grant orphan drug designation to EXG110 highlights the need for better…
Amicus Therapeutics and Teva Pharmaceuticals have struck a deal that will allow the latter to sell a generic version of Galafold (migalastat), Amicus’ chaperone therapy for Fabry disease, to patients in the U.S. Under the agreement, Teva will have its clearance on Jan. 30, 2037, assuming the…
Programs that systematically tested for Fabry disease in nearly 1,000 people with chronic pain at centers in France led to a diagnosis in one individual, a new study reports. The low prevalence suggests systematic testing for Fabry in chronic pain patients “does not seem relevant,” researchers wrote. Nonetheless,…
An international team of scientists has developed a novel algorithm that may help identify people with Fabry disease based on insurance claims data. The model showed particular promise for detecting potential Fabry patients who are young and/or female, according to the study “Unveiling the untreated: development…
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