MIAMI, Fla. -- One in 40,000 children are born with a rare genetic disorder that robs them of their vision. Now, surgeons are performing an innovative gene therapy treatment that is restoring the gift of sight within days!
Ro'Nylah Cummings loves tumbling, but just a year ago she couldn't see an inch in front of her face.
"She would walk right into a wall," said Rolanda Parker, Cummings' mom.
The five-year-old suffered from a rare disorder that would leave her completely blind.
Byron Lam, MD, an Ophthalmologist at Bascom Palmer Eye Institute said, "Lebert Congenital Amaurosis is a congenital retinal degeneration."
Now doctors are performing an FDA- approved procedure that replaces the mutated gene. It's called Luxturna by Spark Therapeutics. First, the surgeon creates a retinal detachment.
"And under the retina you put a virus that has the gene that is defective in her," explained Audina Berrocal, MD, a Pediatric Retina Surgeon at Bascom Palmer Eye Institute.
The virus infects the retinal cells producing the normal protein that restores vision.
Dr. Berrocal said, "Patients even two days after surgery are noticing and doing things that they haven't been able to do before."
Cummings had both eyes done in October 2018. Her mom noticed a difference as soon as they got home!
"We did like four pages of homework together and she could see it just fine and I'm like wow!" Parker stated.
"For me as a surgeon it's incredible," Dr. Berrocal said.
But the price tag for the procedure is hefty.
Dr. Lam said, "The treatment is $425,000 per eye."
But knowing her daughter can now see the family pet...
"My dog's name is Sweetie," Cummings said.
The procedure is priceless.
Parker shared, "It's given me so much hope for her."
So Cummings can keep on dancing.
Her mom tells us Cummings can even see at night now! In some cases, insurance covers part or most of the cost. Doctors say the treatment seems to have a more dramatic impact on children than adults with the same condition. But they say the therapy opens the door for future gene therapies and that may drive down the cost. Follow up studies are only three years out so the long- term benefits of Luxturna are unknown.
Health Watch: Gene Therapy Restores Ro'Nylah's Sight!
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