European CHMP recommends approval of gene therapy (Libmeldy) for the treatment of children with the ‘late infantile’ or ‘early juvenile’ forms of metachromatic leukodystrophy (MLD)

People with MLD have a fault in the ARSA gene, resulting in lack of ARSA enzyme and accumulation of sulfatides in the brain and nervous system. Libmeldy consists of the child’s own stem cells which have been modified to contain working copies of the ARSA gene.

Source:

European Medicines Agency