Brave Girl, 6, Gets Sight Back with Help of Special Therapy

Saffie Sandford got her sight back with NHS eye gene therapy.

A brave 6-year-old girl got her sight back with 'incredible' NHS eye gene therapy.

Saffie Sandford has a rare condition that affects her eyes, called Leber’s Congenital Amaurosis.

Babies with this condition have low vision in daylight and no vision in low light. They can lose their sight completely when they grow up.

Saffie was diagnosed with this condition when she was 5 because she had trouble seeing in the dark.

Saffie has a rare eye condition called Leber's Congenital Amaurosis.

Moorfields Eye Hospital diagnosed Saffie, then she went to Great Ormond Street Hospital for eye gene therapy.

This therapy is the first of its kind for one of the genetic causes of LCA and gives the eye a healthy copy of the gene.

Saffie's mom said they were shocked by the diagnosis but relieved to find a treatment on the NHS.

They were happy to hear there was a treatment for Saffie's condition on the NHS.

Without treatment, Saffie would be blind by 30. Before treatment, she was blind in the dark, which made daily life hard.

Saffie had eye gene therapy in April 2025 and September. She already wore glasses because she was short-sighted.

Saffie got the eye gene therapy, Luxturna, at Great Ormond Street Hospital.

Her mom said the gene treatment was life-changing, like a magic wand restoring her sight.

Now Saffie can go trick-or-treating and to restaurants at night. Her daytime vision has also improved.

Saffie can see hazards and does better in school. She can play like a normal 6-year-old.

Saffie is thriving, and the results are incredible. Her family is grateful for the chance to have her sight back.

Saffie can now see hazards and does better in school.

Researchers found that Luxturna can improve sight and visual pathways in young children.

The study followed 15 children who got the gene therapy between 2020 and 2023.

Doctors treated children from 15 months to 12 years old. The youngest children improved more because they got treatment during a critical period.

The team used a painless test to measure how well signals travel from the retina to the brain.

The researchers are hopeful for good long-term results and will gather more data.

A doctor said they can show that gene therapy strengthens visual pathways in babies and young children with this rare eye condition.

Even small improvements in a child's vision can make a big difference for families.

This research highlights the potential of gene therapy for children with inherited retinal disease.

A new test could set a standard for how future pediatric gene therapy trials are assessed.

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