‘Gene therapy is a game changer for our son’
A five-month-old becomes the first person in England to get a drug with a list price of £1.79m. …
“She couldn’t do the basic things. She couldn’t reach for a toy, which was five centimetres away. We couldn’t leave her alone, Even on the sofa, where she was all surrounded by cushions and pillows because everything was too dangerous,” she says. “We couldn’t relax for a second, and she couldn’t enjoy her life.”
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