Story perspectives

New Gene Therapy Transforms Vision for Young Girl

4/23/2026

Summary

Story summary

Saffie Sandford, a six-year-old from Stevenage, received Luxturna at Great Ormond Street Hospital (GOSH) for Leber's Congenital Amaurosis (LCA).
Her mother described the treatment as transformative, noting Saffie can enjoy trick-or-treating and climbing.
Research from GOSH and UCL shows Luxturna enhances visual pathways in children treated before age 12, assessed with pattern visual evoked potentials (VEPs), and suggests a new standard for evaluating pediatric gene therapy.

1 of 1

Story summary

Saffie Sandford, a six-year-old from Stevenage, received Luxturna at Great Ormond Street Hospital (GOSH) for Leber's Congenital Amaurosis (LCA).
Her mother described the treatment as transformative, noting Saffie can enjoy trick-or-treating and climbing.
Research from GOSH and UCL shows Luxturna enhances visual pathways in children treated before age 12, assessed with pattern visual evoked potentials (VEPs), and suggests a new standard for evaluating pediatric gene therapy.