Saturday, October 14, 2017 03:00 AM

FDA PANEL SUPPORTS GENE THERAPY FOR KIDS WITH RARE EYE DISEASE

By Steven ReinbergHealthDay Reporter

THURSDAY, Oct. 12, 2017 (HealthDay News) -- A U.S. Food and Drug Administration advisory panel on Thursday recommended approval of a gene therapy that could grant the gift of sight to young people with a rare type of inherited[1] vision loss[2].

The panel members' vote was unanimous. The FDA is not required to follow the advice of its panels, but it usually does.

The treatment, which involves replacing a nonworking gene with a new one, is opening a new world for children and teens[3] with the inherited retinal disease called Leber congenital amaurosis.

"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," said Stephen Rose, chief research officer at the Foundation Fighting Blindness.

For those who have already received the therapy, the treatment has been life-changing.

Eleven-year-old Cole Carper got the treatment when he was 8, according to the ...

News source: MedicineNet

See also: EastRidgeEyeCenter