STAT+: Hemophilia gene therapies arrived after 40 years of struggle. Where are the patients?
STAT
MARCH 13, 2024
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.
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