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162 results for "gene therapy"
A groundbreaking gene therapy called Otarmeni is restoring hearing to children born deaf, and the company is offering it completely free in the U.S. Kids who couldn't hear a lawnmower next to them can now hear whispers within months of treatment.
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A single gene-editing treatment could replace daily cholesterol pills for millions, after early trials showed one infusion cut dangerous LDL levels by 62% for up to 18 months. Scientists used CRISPR technology to modify how the body naturally clears cholesterol from blood.
Scientists are testing a one-time treatment that could protect people from heart disease forever. Early results show dramatic drops in cholesterol after a single infusion.
A second-grader ran a lemonade stand for a week to help fund gene therapy research for his best friend Jack, who has a rare genetic disease. The community rallied around the effort, doubling the $5,000 goal.
Scientists tested a one-time gene therapy that lowered cholesterol up to 62% in patients, potentially replacing daily pills and injections with a single treatment. The breakthrough could transform how millions manage heart disease risk.
A new gene-editing treatment could lower bad cholesterol permanently with just one infusion, eliminating the need for daily medication. Early trial results show a 62% drop in LDL cholesterol that's lasted up to 18 months.
A rare muscle disease that once offered families almost no hope now has multiple new treatments helping kids stay stronger longer. Gene therapies and innovative medications are changing what's possible for children with Duchenne Muscular Dystrophy.
Scientists in West Michigan are creating breakthrough treatments that attack the root causes of ALS rather than just managing symptoms. Their work focuses on fixing the genetic mutations that trigger this devastating disease.
A revolutionary gene-editing treatment from Eli Lilly can slash bad cholesterol by up to 62% with just one dose, and the effects last for years. The breakthrough could transform how we prevent heart disease, America's leading killer.
Scientists developed a gene therapy that could reduce painful monthly eye injections for macular degeneration patients down to just once or twice a year. The breakthrough offers hope to nearly 20 million Americans battling the leading cause of vision loss in older adults.
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A single infusion lowered cholesterol dramatically in a small trial, potentially replacing daily pills with a one-time treatment. If confirmed in larger studies, this could transform heart disease prevention for millions.
A one-time gene-editing treatment lowered dangerous cholesterol levels by 62% in early human trials, offering hope for millions who struggle with heart disease prevention. The therapy produced no serious side effects, marking a major safety milestone.
Scientists at UC San Diego have developed a gene therapy that shields brain cells from toxic proteins linked to dementia, successfully preserving memory in mice. The treatment could offer hope for millions facing Alzheimer's, frontotemporal dementia, and ALS.
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.
Basel Abushawish spent 31 years unable to play sports due to severe hemophilia B. After receiving Canada's first dose of breakthrough gene therapy, he's finally heading to the soccer field.
A 31-year-old London, Ontario man with hemophilia B just received Canada's first one-time gene therapy that could free him from a lifetime of treatments. Basel Abushawish is now dreaming of playing soccer for the first time in his life.
After 25 years of research, scientists have submitted the first application to treat genetic diseases in fetuses before birth. The FDA told them they can skip animal testing because the therapy is already proven safe.
A Kansas City hospital will multiply its capacity to deliver breakthrough cell and gene therapies that can cure diseases with a single treatment. The expansion promises to bring life-changing care closer to home for families across the Midwest.
South Korean researchers discovered a protective protein that naturally shields cartilage from destruction, opening the door to treatments that could halt osteoarthritis instead of just masking the pain. A single gene therapy injection showed lasting benefits in animal studies.
A new gene therapy for Duchenne muscular dystrophy met its main goal in trials, bringing hope to families facing this devastating disease. The treatment successfully helped 28 of 30 patients produce a crucial muscle protein that could slow disease progression.
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