Spencer Knight: Breakthrough in Gene Therapy – Life-Changing Vision Restored for Young Girl
Spencer Knight, Business Development Director at Hartmann Young, shared a post on LinkedIn:
“Breakthrough in Gene Therapy: Life-Changing Vision Restored for Young Girl
Saffie Sandford, a six-year-old girl from Stevenage, was diagnosed with a rare inherited eye condition called Leber’s Congenital Amaurosis (LCA) – a disorder that can lead to complete blindness.
Without treatment, her family were told she could lose her sight by adulthood.
In a groundbreaking procedure at Great Ormond Street Hospital, doctors used a one-off gene therapy called Luxturna, injecting a healthy copy of the faulty gene directly into her eyes.
The results have been remarkable.
After struggling to see in low light for years, Saffie can now see in the dark something her family described as ‘like a magic wand.’
She’s now doing everyday things many take for granted, from playing outside at night to enjoying activities she once had to miss out on.
The treatment has significantly improved her vision and quality of life, especially because it was given at such a young age and highlights the power of gene therapy to transform lives of children with rare genetic conditions.
Incredible to see science making the impossible possible.”
Stay updated with Hemostasis Today.
-
Jun 18, 2026, 19:53Astha Thakkar: What Is the Most Important Test for a Bleeding Disorder?
-
Jun 18, 2026, 19:53Shehab Mohamed: Real-World Evidence on Maternal and Neonatal Outcomes in ITP
-
Jun 18, 2026, 19:51Mawadda Chukr: What 10 Randomized Trials Reveal About Novel ITP Therapies
-
Jun 18, 2026, 19:49Ana Rita Santos: Cost-Effectiveness of Chatbot-Supported Remote Monitoring for Anticoagulation Management
-
Jun 18, 2026, 19:46Angela Fan: Highlighting the Significant Burden Faced by Patients Living With PV
-
Jun 18, 2026, 15:29Santagata Davide: Timely Removal of Retrievable IVC Filters May Reduce VTE Recurrence and Mortality
-
Jun 18, 2026, 15:13Youssef Afifi: The Thrombocytosis–Bleeding Paradox
-
Jun 18, 2026, 15:06Nihala Sharin E A: The Vital Role of Hematology in the Medical Laboratory
-
Jun 18, 2026, 14:59Maeve Crowley: Shining a Light on Venous Thromboembolism Trends in Ireland