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Leber's Disease News

FDA Panel Mulls Gene Therapy for Kids With Rare Eye Disease

Posted 12 Oct 2017 by Drugs.com

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. "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 Associated Press. Afterwards, "I looked up and said, 'What are those light ... Read more

Related support groups: Eye Conditions, Visual Defect/Disturbance, Leber's Disease

Gene Therapy Shows Promise for Inherited Eye Disease

Posted 4 May 2015 by Drugs.com

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 incurable retinal conditions," study leader Dr. Samuel Jacobson, of the University of Pennsylvania's Scheie Eye Institute, said in a U.S. National Eye Institute release. "Even though the current version of the therapy doesn't appear to be the permanent treatment we were hoping for, the gain in knowledge ... is an opportunity to improve the therapy so that the restored vision can be sustained for longer durations in patients," Jacobson added. Scientists started the research in 2007, looking at 15 people ... Read more

Related support groups: Eye Conditions, Retinal Disorders, Visual Defect/Disturbance, Leber's Disease

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