The European Medicines Agency (EMA) has issued a negative opinion on a request to approve a less frequent and higher-dose regimen of Elfabrio (pegunigalsidase alfa), an approved enzyme replacement therapy (ERT) for adults with Fabry disease. The request, submitted by Elfabrio’s developers, Chiesi Global Rare Diseases and Protalix…
News
Noninvasive in vivo confocal microscopy (IVCM) detected signs of nerve damage in the cornea — the eye’s transparent outer layer — in people with Fabry disease, who also had higher levels of inflammatory immune cells in the cornea than healthy individuals, a study found. “IVCM provides parameters that reliably…
Long-term use of the approved oral therapy Galafold (migalastat) can help stabilize heart and kidney function in women and girls with Fabry disease across different disease severity levels, a new study from U.S. and European researchers reports. Nearly three-quarters of the female participants in the two clinical trials…
Inflammation, believed to be a secondary or late complication, may instead be an early and active driver of Fabry disease, according to data from a small study in Spain. Researchers found ongoing immune activation and signs of inflammation in all patients, even in those with little buildup of the…
Enzyme replacement therapy (ERT) may help limit damage to blood vessels in the kidneys of people with Fabry disease, a new study reports. Specifically, the study found that people with Fabry who had been on ERT for longer had higher levels of VEGF-165b. This molecule is known to…
High-sensitivity blood tests to measure troponin — a biomarker of heart cell damage — can be used to rule out significant cardiomyopathy, a heart condition, in people with Fabry disease, according to a recent report. In clinical settings, this could help physicians promptly identify patients in need of more…
Treatment with the experimental gene therapy AMT-191 led to increases in levels of the enzyme whose deficit causes Fabry disease for four patients in an early clinical trial. As a result, enzyme replacement therapy (ERT) was discontinued for all patients. AMT-191 is being developed by Uniqure,…
People with Fabry disease spend an average of six hours on activities related to a single treatment with an enzyme replacement therapy (ERT), including on travel, waiting, infusions, and other tasks, an observational study suggests. About 1 in 5 patients and half of caregivers said they took time…
Treatment with Fabrazyme (agalsidase beta) for Fabry disease was safe and tolerated well over about a year, led to reductions in disease-related biomarkers, and eased symptoms in most patients, according to results of a post-marketing surveillance trial in China. An approved enzyme replacement therapy (ERT) for Fabry disease,…
The main part of a Phase 2 trial testing Acelink Therapeutics‘ AL01211 in men with Fabry disease is now complete, and its developer is reporting that the oral treatment candidate showed a favorable safety profile and led to “robust” reductions in disease biomarkers. Given these positive data, Acelink said…
Recent Posts
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- Finding my tribe at FSIG’s recent Fabry community get-together
- New monthly dosing of Elfabrio approved in EU for some Fabry patients
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- FDA grants orphan designation to new Fabry cell therapy GT-GLA-S03
- Sangamo seeks accelerated US approval of gene therapy for Fabry
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- Idorsia outlines new Phase 3 program for lucerastat in Fabry disease
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