First Child in UK Receives “Life-Saving” Gene Therapy for Fatal Genetic Disorder: “A Huge Moment of Hope”
Teddi, a 19-month-old girl, was the first person in the U.K. to receive a new, potentially “life-saving” gene therapy for metachromatic leukodystrophy (MLD), a fatal genetic disorder, outside of a clinical trial. About six months after treatment, she is “a happy and healthy toddler showing no signs of the devastating disease she was born with,” […]
