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The Independent UK
The Independent UK
National
Jane Kirby

Mother hails gene therapy treatment that restored daughter’s sight as ‘life-changing’

  • 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.

IN FULL

Six-year-old girl has vision restored after first-of-its-kind NHS eye gene therapy

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