News

Hydroxychloroquine Side Effects, Fabry Symptoms May Overlap

Rare side effects caused by hydroxychloroquine, a medication used to prevent malaria and to treat several autoimmune diseases, may mimic some symptoms of Fabry disease, a case series suggests. Hydroxychloroquine (HCQ) toxicity was associated with heart, kidney and muscle problems, as observed in Fabry disease. “A thorough investigation should be performed…

Late-onset Fabry Disease May Be More Common Than Estimated

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…

Mitochondrial TRAP1 Helps Restore Lysosome Function in Early Study

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.

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…