Find uplifting stories about heroes, innovations, and solutions
2 results for "** gene therapy"
A Louisiana man with lifelong sickle cell disease received groundbreaking gene therapy that eliminated his symptoms and opened the door to his dream career as a pilot. His story represents hope for thousands living with this painful genetic condition.
After becoming the first country to approve a revolutionary gene-editing therapy in 2023, the UK is now using Casgevy to treat patients with sickle cell disease and β-thalassaemia. The treatment has shown remarkable results, freeing 97% of sickle cell patients from painful episodes for over a year.