A new guidebook aims to support young adults living with Fabry disease. The Fabry International Network (FIN) developed the guide in collaboration with a coalition of young adults with Fabry disease from around the world. “This guidebook was built with young adults, not simply for them,” Anna…
News
Oral treatment with Galafold (migalastat) helped keep kidney and heart function largely stable over years of follow-up in adults with Fabry disease, according to a real-world U.K. study. The therapy also significantly reduced blood levels of lyso-Gb3, a disease biomarker, in both people starting treatment for the first…
A single dose of an RNA-based gene therapy for Fabry disease produced long-lasting levels of the missing key enzyme and reduced the toxic buildup of fatty molecules linked to the disease, according to new preclinical data from Addition Therapeutics. The findings were presented in a poster at the…
The U.S. Food and Drug Administration (FDA) has agreed to meet with Glafabra Therapeutics to discuss plans for clinical testing of GT-GLA-S03, the company’s gene therapy candidate for Fabry disease. The face-to-face meeting, scheduled for July 16, was arranged through the FDA’s INTERACT program, which allows certain developers…
A team of scientists in Spain has developed a new experimental nanoparticle-based treatment approach that aims to address the root cause of Fabry disease through two distinct biological pathways. The new nanomedicine aims to deliver a working copy of the GLA gene, while also reducing production of Gb3, a…
A new experimental gene therapy may boost the production of alpha-galactosidase A (alpha-Gal A) — the enzyme that’s missing or faulty in Fabry disease — beyond the levels of gene therapies now in development. That’s according to laboratory data from a preclinical study that tested the new gene therapy…
A class of medications commonly used to treat heart and kidney disease, called sodium-glucose co-transporter 2 (SGLT2) inhibitors, appears to be safe in adults with Fabry disease and may offer some benefits for heart health, a study suggested. An analysis of outcomes in 48 patients treated with an SGLT2…
Elfabrio (pegunigalsidase alfa), when given every four weeks, was generally safe and helped most Fabry disease patients maintain stability for up to five years. The therapy was particularly effective for women and those who did not develop antibodies against the replacement enzyme. That’s according to interim results of…
Since its implementation in 2017, newborn screening (NBS) for Fabry disease in Tennessee has sharply increased early diagnosis for baby boys, but continues to miss affected girls, a study found. From 2017 to 2024, statewide screening identified 25 boys with Fabry but no girls. Yet clinical data from roughly…
Children with Fabry disease typically experience long delays before receiving a diagnosis, despite symptoms affecting multiple organs that often emerge in early childhood, a nationwide study in China has found. Overall, a median diagnostic delay of at least four years was seen among the more than 60 children…
Recent Posts
- New guide offers support for young adults living with Fabry disease
- Galafold helps stabilize kidney, heart function in Fabry disease: Study
- Addition’s gene therapy for Fabry yields positive preclinical results
- Guest Voice: How to protect mental health after a devastating diagnosis
- In the French Pyrenees, an entire community shares our burdens