5.4K views · 33 reactions | A baby girl born deaf can hear unaided for the first time, after receiving ground-breaking gene therapy when she was eleven months old at Addenbrooke’s Hospital in Cambridge, she is the first British patient in the world and the youngest child to receive this type of treatment. Read more here - https://orlo.uk/Jk43x NIHR Cambridge Clinical Research Facility | Cambridge University Hospitals NHS Foundation Trust | Facebook

Today, the Food and Drug Administration approved a new gene therapy for Spinal Muscular Atrophy. SMA Type 1 is the number one genetic killer of children 2 and under. This first-time gene therapy, developed here at Nationwide Children's, is allowing kids like Evelyn to have a bright future. Watch her incredible story. | Nationwide Children's Hospital

If you’re considering gene therapy, watch this video to better understand how it’s intended to work in diseases like Duchenne. | Sarepta Therapeutics