Gene therapy is a game changer for our son – BBC News

“She could not do the standard things. She couldnt grab a toy, which was 5 centimetres away. We couldnt leave her alone, Even on the couch, where she was all surrounded by cushions and pillows due to the fact that everything was too harmful,” she says. “We couldnt unwind for a second, and she couldnt enjoy her life.”

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