A 14-year-old who spent most of his life blind can now see after an enterprising Miami doctor had the world’s first topical gene therapy reformulated as eye drops.The medication was recently approved as a topical gel to be rubbed onto skin lesions caused by a sporadic disease that leaves behind severe wounds and scar tissue, sometimes resulting in the fusing together of fingers and toes.The disease, which can also cause scar tissue buildup on eyeballs, belongs to a larger group of rare disorders called epidermolysis bullosa (EB) affecting about one in every 50,000 children.
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