All articles tagged with #metachromatic leukodystrophy
U.S. Secretary of Health and Human Services has approved adding Duchenne Muscular Dystrophy and Metachromatic Leukodystrophy to the newborn screening panel, enabling earlier diagnosis and treatment to improve outcomes for affected children.
The FDA has approved Lenmeldy, the first gene therapy for children with metachromatic leukodystrophy (MLD), a rare genetic disease affecting the brain and nervous system. Lenmeldy is a one-time, individualized single-dose infusion made from the patient’s own stem cells, genetically modified to include functional copies of the ARSA gene. Clinical trials showed that treatment with Lenmeldy significantly reduced the risk of severe motor impairment or death compared with untreated children. The most common side effects are fever and low white blood cell count, and patients should be monitored for potential complications.