Mutations likely to cause Fabry disease, particularly those associated with late-onset disease, were found to be more common in an adult population of U.K. residents than the estimated prevalence of the disease itself, a genetic analysis showed. The findings suggest late-onset Fabry disease prevalence may be higher than…

Activating a mitochondrial protein called TRAP1 can help to improve the functionality of lysosomes, the cellular structures that are defective in Fabry disease, a new study reports. The study, “Activation of mitochondrial TRAP1 stimulates mitochondria-lysosome crosstalk and correction of lysosomal dysfunction,” was published in iScience.

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…