Find uplifting stories about heroes, innovations, and solutions
11 results for "genetic treatment"
Children born with epidermolysis bullosa, a painful genetic disorder causing skin to blister at the slightest touch, now have real treatment options for the first time. Three new FDA-approved therapies are healing wounds that doctors could only bandage before.
A patient in the Netherlands just received a groundbreaking genetic treatment that reprograms their own immune cells to hunt down pancreatic cancer. This first-of-its-kind therapy could transform outcomes for one of the deadliest cancers.
A seven-year-old girl born deaf had everyday conversations with her mother just four months after a single injection delivered a missing gene to her inner ear. Ten patients treated in a groundbreaking trial can now hear sounds they never could before.
Scientists at the University of Trento developed the first gene therapy for 10% of cystic fibrosis patients who had no treatment options until now. The breakthrough could lead to a permanent cure instead of lifelong drug dependence.
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 gene therapy trial has restored hearing in 90% of people born profoundly deaf, with improvements lasting over two years. The treatment worked across ages from infants to adults, offering new hope beyond cochlear implants.
Scientists at Yale University discovered that an inexpensive prescription drug already on pharmacy shelves could ease autism symptoms for people with specific genetic mutations. While it won't help everyone with autism, the breakthrough opens doors for more targeted treatments.
Ten people born deaf can now hear after a single gene therapy injection, with some improvements appearing in just one month. Children showed the most dramatic results, including one seven-year-old who began having everyday conversations with her mom.
A groundbreaking initiative promises to make personalized genetic treatments affordable and accessible for millions of children with rare diseases. The "One to Millions" platform transforms how we develop and approve individualized therapies.
Scientists prevented a life-threatening brain condition in newborn mice using RNA therapy, reducing cases by 67%. The breakthrough could spare thousands of babies from risky brain surgery each year.
Pharmaceutical giant Eli Lilly is ramping up its efforts to develop gene therapies that could restore hearing in people with genetic hearing loss. The move signals growing confidence in cutting-edge treatments that address the root causes of deafness.