They Thought Hemophilia Was a ‘Lifelong Thing.’ They May Be Wrong.
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.