4D Molecular Therapeutics (4DMT) has reached an agreement with the U.S. Food and Drug Administration (FDA) to lift the hold on a U.S.-based clinical trial testing 4D-310, an investigational gene therapy for Fabry disease. The hold was based on safety data from the company’s INGLAXA Phase 1/2 trials.
A review study revealed that those with Fabry disease have a high prevalence of a wide range of sleep problems, with excessive daytime sleepiness being the most common. Reviewed studies also commonly reported insomnia, unrefreshing sleep, periodic limb movements, and REM sleep behavior disorder, or physically acting out dreams…
Messenger RNA (mRNA) treatment restored levels of Gal A enzyme activity in heart cells derived from two Fabry disease patients with severe heart involvement, a study showed. The mRNA therapeutic delivered the correct information contained in the GLA gene to produce a fully functional Gal A enzyme, which is…
More than half of adults with Fabry disease were found to have heart involvement that put them at risk for cardiac events — here, primarily associated with irregular heartbeat and heart failure, according to a new MRI study. While an enlarged heart muscle and scar tissue formation were associated…
Changes in the blood vessels within the eyes of people with Fabry disease may act as reliable, noninvasive biomarkers for disease activity, a new study suggests. But, its researchers say, future studies need to collect data over time to establish a grading system for vascular changes during various stages…
An application seeking approval for PRX-102 (pegunigalsidase alfa), an experimental enzyme replacement therapy for adults with Fabry disease, has been resubmitted to the U.S. Food and Drug Administration (FDA). Protalix BioTherapeutics and its partner Chiesi Global Rare Diseases expect the review of their request, sent in…
Despite disease-related eye changes, visual acuity — the sharpness of a person’s vision, with 20/20 denoting perfect clarity of sight — was “good” in children and adults with Fabry disease, a study showed. Eye involvement was not associated with disease severity in adults with Fabry, and overall vision-related…
Gene therapy ST-920 (isaralgagene civaparvovec) continues to be effective and well-tolerated in adults with Fabry disease, according to the latest data from an ongoing Phase 1/2 clinical trial that’s testing the experimental treatment. Five of the participants treated with ST-920 the longest — up to 15 months, or…
A man with Fabry disease undergoing long-term dialysis to support kidney function and receiving enzyme replacement therapy (ERT) experienced recurrent complications related to blood vessels in his brain, a case study reported. While further investigation into such cases is needed, the researchers recommended that Fabry patients with end-stage…
The risk of severe COVID-19 in people with Fabry disease appears to be driven by immune system function rather than by the genetic disorder itself — “similar to the general population” — a small study concluded. “Immunosuppression therapy in kidney transplant recipients represented the highest risk in this [patient]…
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