A 1-Shot Wonder?

An experimental gene therapy could change the lives of those living with hemophilia A, an incredibly high-maintenance bleeding disorder that requires frequent injections.

A trial in the UK saw 13 hemophilia patients injected with a copy of the gene they were missing at birth, which is responsible for making blood clot. 1 dose of the experimental gene therapy cleared the way for the pilot group to give up other treatments. 9 patients’ results were published in the New England Journal of Medicine.

If the potential 1-shot wonder makes it to market, it could transform care—but cost is “the elephant in the room,” says one of the study’s authors, John Pasi.

The Guardian

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