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Duchenne breakthrough therapy leaves behind pioneering families
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author：comprehensive 2025-09-29 01:06:33 - browse：6165

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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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