GlaxoSmithKline says it has treated a child with Strimvelis, its gene therapy for immune deficiency

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Families of patients are starting advocacy groups, raising money for research, and founding biotech companies to advance cures for rare diseases

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The first-in-the-world case is detailed in Thursday’s New England Journal of Medicine

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So far, gene therapy has only treated rare disorders. Now, for the first time, it has been used to treat a boy with sickle cell disease, a common genetic disease

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A one-off injection could one day lower your cholesterol levels for the rest of your life. People born with natural mutations that disable a specific gene have a lower risk of heart disease, with no apparent side effects. Now a single injection has successfully disabled this same gene in animal tests for the first time

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Gene therapy is about ready to be the next big thing. The prospect of treating diseases by tinkering with DNA has a long history of both promise and frustration. Steady progress means 2017 should be the year the technology finally hits the U.S. market. The problem may be figuring out how to pay for cures

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Spark Therapeutics is within striking distance of a landmark green light from the FDA for its treatment for certain forms of blindness

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There are hundreds of early clinical trials, but only a handful of late-stage ones have reached completion

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