Overview
- Saffie Sandford, 6, from Stevenage, regained functional vision after receiving Luxturna at Great Ormond Street Hospital on the NHS.
- Luxturna is a one-time injection that delivers a working copy of the faulty RPE65 gene into the eye to restore the protein needed for vision.
- Researchers at GOSH and UCL reported strengthened retinal-to-brain visual pathways in 15 children treated between 2020 and 2023, with the biggest improvements in the youngest patients.
- The team assessed outcomes using pattern visual evoked potentials, a painless test that tracks signals from the retina to the visual cortex and could guide future pediatric trials.
- She was treated in April 2025 in one eye and September 2025 in the other, and her family say she can now see in the dark and join evening and playground activities.