Centogene has extended a collaboration deal with Takeda in which the two companies will continue to provide diagnostic services to patients with lysosomal storage disorders — which may help people with Fabry disease to get a correct diagnosis. The partnership was initially established to improve patient access…
Measures of heart function improved one to two years after treatment with 4D-310, an investigational gene therapy for Fabry disease, according to new trial data from 4D Molecular Therapeutics (4DMT), the therapy’s developer. “We are pleased to see 4D-310 continue to consistently demonstrate clinical activity across multiple important…
Elfabrio (pegunigalsidase alfa) is as safe and effective as Fabrazyme (agalsidase beta) and can prevent kidney decline in adults with Fabry disease over two years, according to final data from the BALANCE Phase 3 clinical trial. Infusion-related side effects, in particular, occur significantly less often with Elfabrio than…
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…
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