Modifying a potential gene therapy with a specific antibody increased its efficacy in cells that line blood vessels — cells whose dysfunction underlies cardiovascular problems in Fabry disease — a new study from researchers in Spain and the Netherlands showed. This strategy, tested here in a cellular model of…
Noninvasive quantitative magnetic resonance imaging, or qMRI, accurately detected excess globotriaosylceramide (Gb3), the fatty molecule that accumulates in people with Fabry disease, in the brains of people with the disease, a study reports. “We demonstrated the feasibility and clinical relevance of noninvasively assessing cerebral Gb3 accumulation in [Fabry] using…
Researchers detected a mutation in the GLA gene, which is defective in Fabry disease patients, in people with Parkinson’s disease, a study revealed. Parkinson’s patients who carried this mutation showed signs of Fabry, particularly affecting the heart and nervous system. The findings follow a previous report that…
Pregnancy increased the severity and frequency of pain among women with Fabry disease who lived with moderate pain before becoming pregnant, a study from Austria reported. Further, preeclampsia, or dangerously high blood pressure during pregnancy, occurred three times more often in Fabry women than in the general population, according…
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…
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