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It's like someone waved a magic wand: 6-year-old's sight restored by gene therapy
Saffie Sandford, a six-year-old from Stevenage, received Luxturna gene therapy for Leber's Congenital Amaurosis after testing at Moorfields Eye Hospital and treatment at Great Ormond Street Hospital, with one eye treated in April 2025 and the other in September 2025. A related UCL-GOSH study followed 15 children treated between 2020 and 2023, finding greater improvements in younger children. The NHS described the treatment as life-changing but not a cure.
Why It Matters
The case illustrates the potential of gene therapy to improve vision in inherited retinal diseases and highlights how age and developmental timing can influence outcomes.
Timeline
5 Events
Article publication date covering Saffie Sandford's case and related research
April 22, 2026
The article reporting Saffie Sandford's case and the associated research findings was published on April 22, 2026.
Second Luxturna eye treatment for Saffie Sandford
September 2025
Saffie Sandford underwent Luxturna gene therapy on the second eye at Great Ormond Street Hospital in September 2025.
First Luxturna eye treatment for Saffie Sandford
April 2025
Saffie Sandford underwent Luxturna gene therapy on one eye at Great Ormond Street Hospital after tests at Moorfields Eye Hospital in London, marking the first procedure for this genetic form of LCA for her case.
GOSH/UCL Luxturna study results and follow-up (through 2023)
2023
By 2023, follow-up data showed changes in vision were limited in older children, while the youngest children showed more improvement, suggesting benefit during a critical period of visual development.
GOSH/UCL Luxturna study with 15 children (2020–2023)
2020
Researchers at Great Ormond Street Hospital (GOSH) and University College London conducted a study following 15 children treated with Luxturna at GOSH between 2020 and 2023, tracking outcomes with objective measures including pattern visual evoked potentials.