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

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