atidarsagene autotemcel (Libmeldy)
For treatment of metachromatic leukodystrophy (MLD) characterized by biallelic mutations in the arysulfatase A (ARSA) gene leading to a reduction of the ARSA enzymatic activity:
in children with late infantile or early juvenile forms, without clinical manifestations of the disease
in children with the early juvenile form, with early clinical manifestations of the disease, who still have the ability to walk independently and before the onset of cognitive decline
