News

Early diagnosis and a quick start to enzyme replacement therapy (ERT) with agalsidase alfa eased a patient’s Fabry disease symptoms, preventing them from getting worse for at least six months. That’s according to a new report from China that detailed the case of a man in his 30s,…

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…

A 69-year-old man diagnosed with Fabry disease who developed heart failure, high pulmonary arterial pressure, and heart microcirculation dysfunction saw his symptoms ease after treatment with vericiguat, according to a case reported in China. Vericiguat, which is sold as Verquvo, is an oral medicine that’s approved in the U.S.

Nearly one-third of Danish patients in Fabry disease registries were found to have poor lung function, with obstructive airflow limitation most common among those with severe disease and smokers, a study found. People with Fabry “frequently develop an obstructive airflow limitation,” which is found “not only in smokers, but…

People with Fabry disease have higher-than-normal levels of three inflammatory markers in their blood, according to a recent study. These markers include elevated neopterin and biopterin levels, and an increased kynurenine-to-tryptophan ratio. The study, “Serum Neopterin, Biopterin, Tryptophan, and Kynurenine Levels in Patients with Fabry Disease,”…

A woman in her 20s with Fabry disease was safely treated with agalsidase alfa — a commonly used enzyme replacement therapy (ERT) — during her two pregnancies with no resulting complications, a case study reported. During the first pregnancy, “we did not observe any adverse event on…

Fabry Awareness Month, which is marked every April to draw attention to Fabry disease, is shining a light on the “heroes” who are living with the disease and those who support them. The month’s activities seek to heighten awareness among the general public, as well as among researchers,…

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…

Researchers in England are working toward identifying and addressing barriers to reaching a Fabry disease diagnosis among underrepresented communities, particularly within ethnic minority and low socioeconomic status groups. The project is spearheaded by Richard Steeds, MD, a professor at the University of Birmingham and consultant cardiologist at University Hospitals…

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…