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FDA Panel Considering Gene Therapy For Children With Eye Disease

A Food and Drug Administration advisory panel was ready Thursday to recommend approval for a gene therapy that could bring sight to children with a rare type of inherited vision loss. The treatment Replaces a nonworking gene with a new one is restoring sight to those with the inherited retinal disease called Leber congenital amaurosis. If approved by the FDA advisory panel, the treatment would be on its way to becoming the first gene therapy approved for an inherited disease.
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