Good News Network · 5 hours ago

6 Year Old Saffie Has Her Vision Saved From Rare Form of Blindness Thanks to One-Time Gene Therapy

Saffie Sandford was six years old when a single dose of gene therapy -- delivered directly into each eye -- reversed a rare inherited blindness that had been stealing her vision since birth. Leber's Congenital Amaurosis had left her unable to navigate in the dark or see clearly in daylight, a progressive condition that would have led to complete blindness by early adulthood. After receiving Luxturna at Great Ormond Street Hospital, something shifted: "It's like someone waved a magic wand and restored her sight in the dark," her mother says, describing how Saffie can now go trick-or-treating and see hazards she once walked right into. The therapy worked for seven out of ten children in the trial, offering not just restored vision but restored childhoods -- the simple gift of seeing the world that had always been around them.