A six-year-old girl from Stevenage has had her vision restored through a pioneering NHS gene therapy called Luxturna, which treats a rare inherited condition that would have otherwise led to total blindness.
Saffie Sandford, a six-year-old from Hertfordshire, has become the first patient to receive a “life-changing” gene therapy on the NHS to treat Leber’s Congenital Amaurosis (LCA), a rare genetic condition that causes progressive sight loss. The treatment, known as Luxturna, involved injecting a healthy copy of a specific gene directly into her eyes at Great Ormond Street Hospital to replace a missing protein essential for vision. Saffie’s mother, Lisa, described the diagnosis as a “huge shock” but noted the results of the one-off therapy have been “incredible,” enabling her daughter to participate in evening activities like trick-or-treating for the first time.
“Having the gene treatment has been life-changing, it’s like someone waved a magic wand and restored her sight in the dark,” Lisa said, adding that while the family knows the effects “might not last forever,” Saffie is now thriving at school and in everyday play. This medical milestone coincides with research from University College London suggesting that the therapy is particularly effective at strengthening visual pathways during critical stages of brain development.
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