> Libmeldy is made using stem cells that are derived from a patient's blood or bone marrow and can give rise to different types of blood cells, according to the

> Libmeldy is made using stem cells that are derived from a patient's blood or bone marrow and can give rise to different types of blood cells, according to the European Medicines Agency(opens in new tab) (EMA). These stem cells carry the new, functional genes into the body, where they give rise to white blood cells that travel through the bloodstream. > In clinical trials, Libmeldy offered clear benefits to infantile and juvenile patients who hadn't yet developed MLD symptoms; these patients were able to break down sulfatides at normal rates and showed typical patterns of motor development, for example. The benefit of the therapy seemed to last several years, but at this point, "it is not yet clear whether it will persist life-long, and extended follow-up is needed," the EMA noted. > Libmeldy is approved for use in the European Union and U.K., although the U.K.'s drug price watchdog initially rejected the therapy due to its hefty list price of £2.8 million ($3.4 million at today's exchange rates), BBC News(opens in new tab) reported in 2022. The therapy's manufacturer, Orchard Therapeutics, then offered Libmeldy to the NHS at a significant discount. > The gene therapy has not yet been approved by the U.S. Food and Drug Administration(opens in new tab).