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First Gene Therapy For Children With Metachromatic Leukodystrophy Is Approved By The FDA
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gene therapy FDA approval The U.S. Food and Drug Administration has granted Lenmeldy (atidarsagene autotemcel) approval. The FDA has approved this gene therapy as the first of its kind for the treatment of children with pre-symptomatic late infantile, pre-symptomatic early juvenile, or early symptomatic early juvenile metachromatic leukodystrophy (MLD). Metachromatic leukodystrophy (MLD) Metachromatic leukodystrophy is an uncommon, incapacitating hereditary disorder that specifically …