News

AceLink’s AL01211 Granted FDA Orphan Drug Status for Fabry

AL01211, a once-daily oral therapy being developed by AceLink Therapeutics for Fabry disease, has been granted an orphan drug designation by the U.S. Food and Drug Administration (FDA). The FDA gives this designation to investigational treatments with the potential to improve care for rare diseases that affect fewer than…

European Teens Report High Fabry Burden, Not Fully Lessened by ERT

The Fabry disease burden is variable but significant among adolescents and caregivers, affecting their quality of life and mental health, according to results of online surveys in three European countries. In addition, standard enzyme replacement therapy (ERT) was reported to reduce the disease’s burden, but not fully. These findings highlight…

Patients Wanted to Test Device for Detecting Cardiac Problems in Fabry

A clinical study in the U.K. is recruiting adults with Fabry disease (FD) to determine whether implantable loop recorders (ILRs) — a device for continuous heart monitoring — can improve arrhythmia detection and identify risk predictors for cardiac problems. Participants’ heart rhythms will be tracked, using the surgically implanted…