In this Oct. 4, 2017 photo, Dr. Albert Maguire checks the eyes of Misa Kaabali, 8, at the Children’s Hospital of Philadelphia. (AP Photo/Bill West)
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On Thursday, U.S. Food and Drug Administration advisers will consider whether to recommend approval of a gene therapy that improved vision for these three youths and some others with hereditary blindness.Only one gene therapy is sold in the U.S. now, a cancer treatment approved in August that engineers patients' blood cells in the lab.The treatment, Luxturna, is made by Philadelphia-based Spark Therapeutics.The company's Nasdaq ticker symbol is ONCE, for how often it hopes the therapy is needed.That group was allowed to get the therapy after waiting one year, so in all, 29 were treated, plus more in earlier studies.There were two serious side effects, both deemed unrelated to the gene therapy itself.It took two years for gene testing to determine whether the Carper kids would qualify for the study, and insurance wouldn't pay because there was no established treatment.For Christian Guardino, a senior at Patchogue-Medford High School on Long Island, the most remarkable part about performing on "America's Got Talent" a day before his 17th birthday earlier this year wasn't winning the golden buzzer that showered gold confetti on him and sent him into further competition. It was seeing the confetti thanks to his gene therapy several years ago.RPE65 mutations can cause other vision diseases besides LCA, so if the treatment is approved, it should be for people with the flawed gene rather than a specific disease, said Dr. Eric Pierce at Harvard-affiliated Massachusetts Eye and Ear, who was involved in its early testing.
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