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First Gene Therapy for Children with Metachromatic Leukodystrophy is Approved by the FDA

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 childr...
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FDA approves gene therapy to treat beta thalassemia

FDA approval of gene therapy The FDA has made it possible for people 12 years of age and older who have transfusion-dependent beta-thalassemia (TDT) to use exagamglogene autotemcel (Casgevy, Vertex)....
fda-approved-genetic-treatment

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