questionText: To ask the Secretary of State for Health and Social Care, how many children diagnosed with metachromatic leukodystrophy since the approval of gene therapy treatment have been assessed as ineligible for treatment because diagnosis occurred after symptoms developed.
answerText: Libmeldy, the gene therapy treatment for metachromatic leukodystrophy (MLD) has been funded nationally by NHS England since 2022 and is delivered exclusively through the specialist children’s service at Royal Manchester Children’s Hospital. Information on...
heading: Metachromatic Leukodystrophy