Becker's Healthcare March 19, 2024
On March 18, the FDA approved the first gene therapy for children with metachromatic leukodystrophy, a debilitating, rare genetic disease.
The medicine, Lenmeldy (atidarsagene autotemcel), is a one-time infusion made from the patient’s hematopoietic stem cells and modified copies of the arylsulfatase A gene, the FDA said. The genetic disease, which is caused by an ARSA gene deficiency, manifests into the loss of motor and cognitive function, and early death.
In a trial,...
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