7-Year-Old Receives New FDA-Approved Retina Gene Therapy – University of Michigan Health System News
Posted: September 19, 2019 at 3:37 pm
Kari Branham, M.S., a genetic counselor at Kellogg, worked with Zions family to help them understand the genetic basis for Zions condition.
SEE ALSO: Retinitis Pigmentosa in Children: 5 Facts Families Should Know
We have seen such amazing progress with these conditions over the last 15-20 years,says Branham. We used to tell patients and their families that we would have to wait and see what happens, but now we can actually do something to help.
By going through the gene therapy process, Branham says the team is hopeful that this has changed Zions prognosis.
The treatment is designed to stop or slow the death of specialized cells in the retina, called photoreceptors, that send visual information to the brain.
Patients who have Leber congenital amaurosis have night blindness, says Besirli. One of the first treatment effects after receiving Luxturna is that (patients) are telling us that they function much better in dark.
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They can play outside much longer, they can navigate around the house and dont need nightlights anymore and can participate in indoor sports. Thats been a huge change in their lives.
Seven months after treatment, Zion, now age 7, and his family are back to their normal routine in Montrose, Mich., and monitor his progress during follow-up appointments at Kellogg.
Zion says hes looking forward to playing football and, with improved vision -- playing outside at night with his brothers.
We hope that with Zion we have changed the trajectory for him to the point that in his 20s he wont have significant vision loss we see with him now, says Branham.
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7-Year-Old Receives New FDA-Approved Retina Gene Therapy - University of Michigan Health System News
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