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…
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FLT190, an experimental gene therapy from Freeline Therapeutics, safely led to stable increases in the activity of alpha-galactosidase A (alpha-gal A) — the enzyme that is missing in Fabry disease — and prevented the buildup of globotriaosylceramide (Gb3) and lyso-Gb3 in a mouse model of the disease.
Adults with Fabry disease exhibited high levels of adherence to at-home Galafold (migalastat) treatment over a two-year period, according to a recent study. Patients also reported a stable quality of life, with reductions in pain and physical limitations over time — all of which are factors that can influence…
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…
A program that screened nearly 500 men with implanted pacemakers for Fabry disease found that none of them had the genetic disorder. The findings “suggest that the prevalence of [Fabry disease] in male patients requiring permanent pacing is very low and does not justify a systematic screening,” according to…
Male patients with Fabry disease who started treatment with Fabrazyme (agalsidase beta) at an earlier age had a slower decline in kidney function compared with older patients with more severe disease, according to a study assessing data from the Fabry Registry. Continued treatment in female and male patients…
Note: This story was updated Dec. 12, 2022, to correct the date by which the FDA must announce its decision about whether to approve PRX-102 is May 9, 2023. The U.S. Food and Drug Administration (FDA) has accepted for review a second biologics license application (BLA)…
Daily treatment with the investigational oral therapy venglustat in men with Fabry disease led to significant reductions in globotriaosylceramide (Gb3 or Gl-3) — the fatty molecule that accumulates to toxic levels in the disease — and no signs of disease progression. That’s according to up to three years…
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…
Genetic screening for 331 family members of Russian Fabry disease patients led to the detection of 165 undiagnosed cases, a study reports. Family genetic screening was also found more effective than other screening programs for identifying Fabry cases. “Family genetic testing was significantly more effective (49.8%) than screening programs…
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