Novel gene therapy reduces risk of motor impairment in MLD
The FDA has approved Lenmeldy (atidarsagene autotemcel) as the first gene therapy for children with presymptomatic late infantile, pre-symptomatic early juvenile, or early symptomatic early juvenile metachromatic leukodystrophy (MLD). MLD is a rare genetic disease affecting the brain and nervous system due to a deficiency of the enzyme arylsulfatase A (ARSA), leading to sulfatide buildup and neurological damage. Lenmeldy is a one-time infusion of the patient’s own genetically modified hematopoietic stem cells, providing functional copies of the ARSA gene to help break down harmful substances. Before treatment, patients undergo chemotherapy to prepare for cell transplantation. In a clinical trial, Lenmeldy significantly reduced the risk of severe motor impairment or death compared to untreated participants, with all presymptomatic late infantile MLD participants alive at 6 years and 71% of treated participants walking unaided at 5 years. Treated participants also demonstrated improved language and cognitive scores compared to untreated peers. The approval of the gene therapy marks a significant milestone in MLD treatment, offering hope for improved outcomes and potentially altering disease progression.
Source: asamonitor.pub/3U5WhCj
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