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…
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…
ISU303, an experimental enzyme replacement therapy for Fabry disease, showed a good safety profile and some promising signs of efficacy in a small clinical trial in Asia, according to researchers. The “results suggest that ISU303 is safe and effective and [may be an] alternative ERT for [Fabry disease],” the…
One-time treatment with ST-920 (isaralgagene civaparvovec), an experimental gene therapy being developed by Sangamo Therapeutics, continues to be generally well-tolerated among people with Fabry disease, according to new data from the Phase 1/2 STAAR clinical trial. Trial data show that the gene therapy leads to long-term increases in activity…
Rare side effects caused by hydroxychloroquine, a medication used to prevent malaria and to treat several autoimmune diseases, may mimic some symptoms of Fabry disease, a case series suggests. Hydroxychloroquine (HCQ) toxicity was associated with heart, kidney and muscle problems, as observed in Fabry disease. “A thorough investigation should be performed…
The first patient has been dosed with the second, higher dose of FLT190, Freeline Therapeutics’ investigational gene therapy for Fabry disease that’s being studied in its Phase 1/2 MARVEL-1 clinical trial. Freeline’s decision to proceed with the higher dosing level of 1.5×1012 vector genomes per kilogram of body…
Implementing nationwide screening programs for Fabry disease in people with a heart condition called hypertrophic cardiomyopathy (HCM) may help improve diagnosis of the disease, a study in Czech Republic shows. In the country, the program led to the diagnosis of Fabry in four men and two women among the…
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