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lebers congenital amaurosis

October 21, 2017

An FDA advisory panel recommends approval for Luxturna, a novel gene therapy for the treatment of two inherited retinal diseases which cause blindness.  Both Leber's congenital amaurosis and retinitis pigmentosa 20 are rare types of retinal blindness caused by a defective RPE65 gene. The FDA will not officially consider approval of Luxturna until January...