A Family's $4 Million Bet on a Groundbreaking Gene Therapy
Jo Kaur describes how her family created a patient‑driven initiative to develop a first‑in‑human gene therapy for their son with Cockayne syndrome, raising about $4 million to bring the treatment from concept to clinic and weighing whether to proceed with the procedure; three months post‑treatment, their son is clinically stable and showing encouraging signs, though long‑term benefits are still uncertain and the family aims to secure FDA approval to treat other children.