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...