STAT+: A bellwether moment: Once a distant dream, gene therapy for Duchenne nears historic decision
For Sarepta, an experimental gene therapy is a story of foresight, savvy dealmaking, scientific puzzle solving, and perseverance.
Brent and Sabina Furbee could see something was wrong with their son, Emerson, even if the doctors didn’t. They were the kind of signs easily dismissed: falling at music class, struggling to climb a ladder at the playground.
A physical therapist near their home in rural Tennessee said the 3-year-old had flat feet and prescribed ankle braces. “I wish I was kidding,” said Brent.
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