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STAT March 18, 2024
The Food and Drug Administration on Monday approved a gene therapy for metachromatic leukodystrophy, a devastating genetic disorder that eats away at affected children’s neurons.
The medicine, marketed as Lenmeldy, is the first treatment for MLD, which is otherwise fatal. Lenmeldy has to be given before symptoms appear or, in some cases, after the first signs of disease, so it won’t be helpful for many patients...
