The New England journal of medicine
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Editorial Comment
Long-term effects of retinal gene therapy in childhood blindness.
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Multicenter Study Clinical Trial
Improvement and decline in vision with gene therapy in childhood blindness.
Retinal gene therapy for Leber's congenital amaurosis, an autosomal recessive childhood blindness, has been widely considered to be safe and efficacious. Three years after therapy, improvement in vision was maintained, but the rate of loss of photoreceptors in the treated retina was the same as that in the untreated retina. Here we describe long-term follow-up data from three treated patients. Topographic maps of visual sensitivity in treated regions, nearly 6 years after therapy for two of the patients and 4.5 years after therapy for the third patient, indicate progressive diminution of the areas of improved vision. (Funded by the National Eye Institute; ClinicalTrials.gov number, NCT00481546.).
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In 2012, a total of 32 organizations entered the Pioneer accountable care organization (ACO) program, in which providers can share savings with Medicare if spending falls below a financial benchmark. Performance differences associated with characteristics of Pioneer ACOs have not been well described. ⋯ Year 1 of the Pioneer ACO program was associated with modest reductions in Medicare spending. Savings were greater for ACOs with higher baseline spending than for those with lower baseline spending and were unrelated to withdrawal from the program. (Funded by the National Institute on Aging and others.).