Experimental gene therapies have yielded promising results in early trials. But the drugs have left some patients worried that success will not last. Scientists are edging closer to defeating a longtime enemy of human health: hemophilia, the inability to form blood clots. After trying for decades to develop a gene therapy to treat this disease, researchers are starting to succeed. In recent experiments, brief intravenous infusions of powerful new treatments have rid patients — for now, at least — of a condition that has shadowed them all their lives.
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