Spiderman-loving Saffie was diagnosed with LCA when she was five and a half-years-old after her parents noticed she was struggling to see in the dark.
After undergoing tests at Moorfields Eye Hospital, she was transferred to GOSH to have the eye gene therapy, Luxturna, which is the first of its kind for one of the genetic causes of LCA.
Mum Lisa said: “Saffie’s diagnosis came as a huge shock to us, as we’d never heard of the condition or knew me and her dad Tam were carriers. It was such a rollercoaster of a journey, but we were so relieved and grateful when we heard there was a treatment available on the NHS for Saffie. We were told that without the treatment, she would be blind by the age of 30.
Saffie had the eye gene therapy in her first eye in April 2025 just before her sixth birthday, and in her second eye in September 2025.
“Having the treatment has been life-changing, it’s like someone waved a magic wand and restored her sight in the dark. We’ve been able to take her trick or treating, and out to restaurants in the evening – something that was impossible before.
“Her peripheral sight in the daylight has also improved. She’s now able to see hazards and has improved at school. She’s thriving and you wouldn’t know she had the condition just by looking at her.”
deran6ed on June 1st, 2026 at 14:05 UTC »
Her: Dad I don't want to be a Marvel character
Dad: shut up Callisto! You will see in the dark and you will like it!
whogotthekeys2mybima on June 1st, 2026 at 13:34 UTC »
I owned stock in Spark Therapeutics when they were bought out years ago and got a pretty penny for it. I love investing in treatments that help cure people. Unfortunately that’s not always the case with gene therapies. Biotech loves treatments that need to be given in perpetuity rather than cures. They’d rather have money than cure people sometimes, and I despise that
jimbobicus on June 1st, 2026 at 13:21 UTC »
Gene therapy seems pretty extreme when they could have just turned on the light instead