ARSA and metachromatic leukodystrophy: Atidarsagene autotemcel (registered as Libmeldy®—hereafter referred to as ‘arsa‐cel’), an autologous HSC‐GT product based on autologous CD34+ stem cells transduced ex‐vivo with a lentiviral vector encoding the human ARSA gene, has demonstrated sustained clinically relevant benefits in children with early onset MLD, by preserving cognitive and motor function, and slowing demyelination and brain atrophy.15, 16