The European Medicines Agency (EMA) has validated a regulatory application that seeks clearance of a less frequent regimen of Elfabrio (pegunigalsidase alfa), an approved enzyme replacement therapy (ERT) for adults with Fabry disease. If the agency decides to approve the variation application after its review, Fabry patients in…
A wide array of symptoms that affected the kidneys and cardiovascular and nervous systems led to a diagnosis of Fabry disease for a 32-year-old woman, a study in India shows. Given that Fabry has been historically considered a male disease and that women carrying a Fabry-causing mutation may not…
The National Organization of Rare Disorders (NORD) is offering financial assistance to people with Fabry disease and their caregivers who may be facing challenges in managing treatment and care-related expenses not covered by health insurance. The nonprofit’s initiatives fall under its RareCare patient assistance programs, which were…
People with Fabry disease have higher levels of certain inflammatory cytokines — small proteins that act as chemical messengers of the immune system — in their blood than do healthy individuals, according to a study by researchers in China. Higher blood levels of some of these inflammatory proteins were…
Get regular updates to your inbox.