THINK MLD, TEST EARLY, REFER URGENTLY AND SCREEN SIBLINGS
Every day counts with MLD
Libmeldy: Ex vivo autologous HSC gene therapy for early-onset MLD
Libmeldy is an autologous haematopoietic stem cell (HSC) gene therapy product for early-onset MLD made specifically for each patient using the patient’s own blood stem cells.1
What is MLD?
Metachromatic leukodystrophy (MLD) is a rare, inherited, neurometabolic disease that causes rapid and progressive demyelination and neurodegeneration, leading to severe neurological disability and death.2 MLD is caused by mutations in the arylsulfatase A (ARSA) gene which lead to ARSA enzyme deficiency.1
Libmeldy is indicated for the treatment of children with early-onset MLD. CD34+ cells are transduced ex vivo with a lentiviral vector encoding ARSA cDNA. It is an autologous gene therapy, meaning a patient’s own stem cells are genetically corrected by inserting a functional copy of the ARSA gene and these are returned to their body.1
▼ This medicinal product is subject to additional monitoring.
Healthcare professionals are asked to report any suspected adverse events. Further information about local reporting details can be found in Section 4.8 of the Summary of Product Characteristics.