Marisa Wexler, MS, senior science writer —

Freeline Therapeutics is pausing development of FLT190, an experimental gene therapy for Fabry disease. The move will let the company free up funds to focus on advancing FLT201, a gene therapy being developed to treat another genetic disorder called Gaucher disease. “While we remain encouraged by the…

The same mutation caused both late-onset and classic forms of Fabry disease among different members of the same family, according to a new report. “To our knowledge, there are no previous reports showing that the same missense mutation causes both late-onset and classic form of [Fabry disease] in male…

Testing family members of people diagnosed with Fabry disease can identify new individuals with the condition and improve their health outcomes, a recent study highlights. “Family screening is of great significance in finding new patients with [Fabry disease],” the researchers wrote. “Therefore, genetic counseling should be recommended to all…

The European Commission (EC) is expected to decide whether to approve PRX-102 (pegunigalsidase alfa) for adults with Fabry disease in early May. The therapy’s co-developers, Protalix BioTherapeutics and Chiesi Global Rare Diseases announced that PRX-102 received a positive opinion from the Committee for Medicinal Products for…

People with Fabry disease often have impairment in the nerves that help regulate heartbeat, which may serve as a prognostic measure to assess heart problems in the disease, a study reports. Mild problems with heart nerves were found in many patients in the disease’s earlier stages who didn’t have…

More than one in 10 people with Fabry disease have a form of swelling called lymphedema, a recent study reported that highlighted the importance of screening for this type of swelling, which often goes unrecognized. “Strategies to identify lymphedema in a timely manner can facilitate effective treatment and minimize…

The U.S. Food and Drug Administration (FDA) has placed a hold on the 4D-310 clinical program, an experimental gene therapy for Fabry disease being developed by 4D Molecular Therapeutics (4DMT). The hold was disclosed in a filing submitted by 4DMT to the Securities and Exchange Commission earlier this month.

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…

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…