Saffie Sandford's Luxturna Gene Therapy Success

Saffie Sandford, a six-year-old girl, had her sight restored thanks to a life-changing eye gene therapy called Voretigene neparvovec on the United Kingdom — National Health Service. Saffie Sandford suffers from Leber congenital amaurosis, a rare inherited condition that prevents normal vision. Moorfields Eye Hospital diagnosed her, and Great Ormond Street Hospital administered the Voretigene neparvovec treatment, which involves injecting a healthy copy of the gene directly into the eye. Researchers at Great Ormond Street Hospital and University College London have published research showing that Voretigene neparvovec can improve sight and strengthen visual pathways in young children, though it is not described as a cure. The treatment has significantly improved Saffie Sandford's quality of life, allowing her to see in the dark and improving her peripheral vision.