STAT+: Hemophilia gene therapies arrived after 40 years of struggle. Where are the patients?
A cautionary tale: researchers spent billions of dollars developing gene therapies for hemophilia. But now it looks like many patients don't want them.
As 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.
“I’m on the fence,” he said.
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