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LENMELDY is a one-time gene therapy developed to treat children with pre-symptomatic late infantile, pre-symptomatic early juvenile and early symptomatic early juvenile, referred to as early-onset, metachromatic leukodystrophy (MLD). MLD is caused by a defect in the arylsulfatase A (ARSA) gene, which causes the body to produce reduced or no ARSA enzyme. LENMELDY is made specifically for each child, using the child’s own blood stem cells, and adding functional copies of the ARSA gene to their cells. This may allow you to produce sufficient ARSA enzyme to stop or slow the progression of MLD symptoms.