University of Florida

Karen Wright worked her way through the small gathering outside her 4-month-old daughter’s room in pediatric intensive care. It seemed to her as if everyone at UF Health Shands Children’s Hospital had come to witness this historic moment. Wright leaned down and whispered to her baby, “This drug is going to save your life.” Londyn became the first baby in the country treated with a newly approved gene therapy for a rare and debilitating neuromuscular disease since it won federal approval in May.