- Six-year-old Saffie Sandford, from Stevenage, has had her sight restored thanks to pioneering eye gene therapy on the NHS.
- Saffie was diagnosed with the rare inherited condition Leber’s Congenital Amaurosis (LCA), which severely affects vision, particularly in low light.
- The treatment, called Luxturna, involves injecting a healthy copy of a gene directly into the eye, and was administered at Great Ormond Street Hospital (Gosh).
- Her mother, Lisa, described the therapy as "life-changing," enabling Saffie to participate in activities like trick-or-treating and improving her peripheral vision and school performance.
- Research by Gosh and University College London indicates that Luxturna can improve visual pathways in young children with RPE65-related retinal disease, with earlier treatment showing greater benefits.
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