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Wider Roll Out of Blindness Gene Therapy

The new gene therapy for childhood blindness has gone into wider use, reports Stat News. It follows the case of 13-year-old Jack Hogan, who has retinal dystrophy and who received the treatment in one eye on Tuesday.

The US Food and Drug Administration approved the therapy, Spark Therapeutics' Luxturna, in January. It involves injected each of the patients' affected eyes with viruses that carry normal versions of RPE65 gene. In a clinical trial, the company said the treatment led to improved vision in 93 percent of patients. However, the treatment is expensive, with a $425,000 per eye price tag.

Stat News reports that Jack Hogan's family held off on getting him the treatment until the FDA approved it, as Jack still had some sight and they didn't want to risk him going blind because of an unproven drug. But it adds that his parents anxiously followed its development. Stat News says Jack's treatment has gone off largely without a hitch, though there was a last-minute scramble to assure his insurance company that Jack's RPE65 gene mutation was the cause of his disease. Now, it adds, they are just waiting to see whether it worked for him and to have his other eye treated.