Doctors took his stem cells, corrected a faulty mutation within them, and used them to replace 80 percent of his skin

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Who will pay for it? And how?

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A one-time gene therapy that costs half a million dollars sounds crazy until you add up what it costs to treat some diseases over a lifetime

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The first gene therapy treatment in the United States was approved recently by the Food and Drug Administration, heralding a new era in medicine that is coming faster than most realize — and that perhaps few can afford

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The disease may be among the first to be treated with the novel gene-editing tool

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How an unusual medical case in the 1990s provided a clue for how to treat a fatal muscle disease

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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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