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Leber's Disease News

FDA Panel Mulls Gene Therapy for Kids With Rare Eye Disease

Posted 12 Oct 2017 by

THURSDAY, Oct. 12, 2017 – A U.S. Food and Drug Administration advisory panel was poised on Thursday to recommend approval for a gene therapy that could grant the gift of sight to young people with a rare type of inherited vision loss. Replacing a nonworking gene with a new one is opening a new world for children and teens with the inherited retinal disease called Leber congenital amaurosis. ...

Gene Therapy Shows Promise for Inherited Eye Disease

Posted 4 May 2015 by

SUNDAY, May 3, 2015 – A new study finds that gene therapy quickly improves eyesight for patients who've lost their vision from an inherited condition called Leber congenital amaurosis (LCA). But the improvements aren't permanent: Researchers said the gains began to diminish after one to three years. "Gene therapy for LCA demonstrated we could improve vision in previously untreatable and ...

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