New disease modifying therapies for two genetic childhood-onset neurometabolic disorders (metachromatic leucodystrophy and adrenoleucodystrophy).

When the modified cells are injected into the patient as a one-time infusion, the cells are expected to produce the ARSA enzyme that breaks down the build-up of sulfatides in the nerve cells and other cells of the patient's body. This holds great promise for the patients and their carers since both diseases are associated with a shortened life expectancy and a heavy disease burden. Treatment with Libmeldy should only be carried out in qualified specialised treatment centres, and patients and their carers should receive extra educational material to warn them of the symptoms of leukaemia and lymphoma. [Extracted from the article]