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How many hemophilia patients actually want gene therapy?

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A s a boy growing up with hemophilia A, Noah Frederick reserved the end of his annual checkups to talk about new technologies. His doctor walked through various experimental approaches for the bleeding disorder and, invariably, gene therapy. It was coming, he always said, in your lifetime. Last year, it finally arrived. But Frederick, now a 23-year-old software engineer, isn’t sure he’ll get it. He wants to be part of the sci-fi future gene therapy has always represented, but a recent doctor’s visit left him uncertain. advertisement “I’m on the fence,” he said. He and, it seems, thousands of other patients. For four decades, researchers and companies searched for ways to replace the broken blood-clotting genes that cause hemophilia, a multibillion dollar effort designed to turn a chronic, sometimes debilitating disease into a curable one. Understand how science, health policy, and medicine shape the world every day But the first two gene therapies have so far been met with crickets. On

A s a boy growing up with hemophilia A, Noah Frederick reserved the end of his annual checkups to talk about new technologies. His doctor walked through various experimental approaches for the bleeding disorder and, invariably, gene therapy. It was coming, he always said, in your lifetime. Last year, it finally arrived. But Frederick, now a 23-year-old software engineer, isn’t sure he’ll get it. He wants to be part of the sci-fi future gene therapy has always represented, but a recent doctor’s visit left him uncertain. Advertisement “I’m on the fence,” he said. STAT+ Exclusive Story Already ha

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