FDA Panel Supports Gene Therapy for Kids With Rare Eye Disease

Thursday, October 12, 2017

A U.S. FDA advisory panel recommended approval of a gene therapy that could grant the gift of sight to young people with a rare type of inherited vision loss. The treatment, which involves 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. FFB chief research officer, Steve Rose, said "This is a gene therapy that can restore some vision to people who have very limited vision or no vision due to the mutation in the RPE65 gene, and as such, it's a great breakthrough." See what else Steve had to say in Health Day News here.