Galafold may reduce heart changes, boost exercise tolerance

In previously untreated Fabry disease patients with heart involvement, 18 months of treatment with Galafold (migalastat) stabilized measures of heart disease and was linked to a trend toward improvement in exercise tolerance, an observational study reported These findings provide “new detailed evidence on the effect of migalastat on…

Gene Therapy 4D-310 Boosts Heart Health in 3 Patients After 1 Year

One year after treatment with 4D-310, an experimental gene therapy for Fabry disease, measures of heart health have improved in the first three participants in a clinical trial, according to new data announced by the therapy’s developer, 4D Molecular Therapeutics (4DMT). “4D-310 holds promise as a potential first-in-class treatment…

CARS Microscopy Technique May Help Identify Heart Involvement

A tissue analysis technique called CARS microscopy could be useful for identifying heart involvement in Fabry disease, according to a new study. Fully known as Coherent anti-Stokes Raman microscopy, the technique “allows early, reliable and efficient detection of [Fabry]-triggered alterations in affected organs,” such as the heart, the researchers…

Fabry International Network Seeks Entries for FIN Award 2022

The Fabry International Network (FIN) is calling for global submissions for its annual FIN Award, which recognizes the efforts of patient organizations to educate and unify the Fabry disease community and to heighten awareness of the rare genetic lysosomal storage disorder. Projects must be initiated this year for…