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A New Jersey teen became the first patient in the state to be cured of sickle cell disease using his own genetically modified cells, eliminating the need for a donor. The breakthrough offers hope to thousands who lack family matches.
Nemours Children's Hospital in Wilmington can now offer a groundbreaking one-time gene therapy that could give children with sickle cell disease a chance at a transformed life. The FDA-approved treatment addresses the root genetic cause of the disease that affects 100,000 Americans and shortens life expectancy by more than 20 years.
A 24-year-old who lost his sister to sickle cell disease just became the first Michigan patient cured by groundbreaking gene therapy. Chantez Sanford is heading back to college healthier than he's ever been.
Jessica Ceja became the first person in Maryland to receive groundbreaking gene therapy that could cure her lifelong sickle cell disease. Her own cells were reengineered in a lab to produce healthy red blood cells, potentially ending decades of pain.
A groundbreaking partnership will bring one-time gene therapies for sickle cell disease and beta-thalassemia to Saudi Arabia and the Middle East, where over 500,000 people live with these genetic blood disorders. Local manufacturing will make these life-changing treatments more accessible to patients who desperately need them.
A 41-year-old mother who spent her life battling sickle cell disease just became the first adult in Maryland to receive a potentially curative gene therapy using her own cells. After decades of hospital stays and missing family moments, Jessica Ceja now has hope for a life without the painful disease.