Find uplifting stories about heroes, innovations, and solutions
25 results for "genetic disorder"
Children with rare genetic growth disorders grew twice as fast after starting a groundbreaking treatment, offering hope where few options existed before. The therapy worked especially well for kids whose condition directly matched the drug's target.
Researchers discovered how to lower dangerous cholesterol without relying on damaged receptors, offering hope to 1 in 200 adults with a common genetic disorder. The breakthrough uses human liver cells to test compounds that stop cholesterol particles from forming in the first place.
A young cancer patient with a rare genetic disorder can now receive groundbreaking immunotherapy treatment in his hometown instead of traveling hundreds of miles to Dallas. The new option comes as Lubbock opens a major cancer center, bringing big-city care to rural West Texas.
People with a rare genetic disorder that causes dangerous fat buildup in their blood now have a breakthrough treatment that cuts triglyceride levels by nearly a third. The UK just approved olezarsen for adults with familial chylomicronemia syndrome, a condition that can trigger life-threatening pancreas inflammation.
A mother whose daughter was diagnosed with a rare genetic disorder created an AI platform that saves families 53 hours a week and accelerates treatment research by up to 50%. Citizen Health now helps over 8,000 patients across 350 rare diseases navigate care and connect with others facing the same challenges.
Twenty-seven out of 28 patients with severe sickle cell disease are now living pain-free after a groundbreaking gene editing treatment. The one-time therapy is offering new hope to people living with a genetic disorder that typically shortens life expectancy to the mid-40s.
Two sisters born with a fatal genetic disorder are thriving thanks to breakthrough treatments that didn't exist a decade ago. Their family's story shows how medical innovation is transforming once-hopeless diagnoses into lives full of possibility.
After losing their 3-year-old son George to a rare genetic disorder, an Akron couple turned grief into action by creating the Be Cheechy Foundation. More than 2,400 people have already been "cheech'd" with random acts of kindness.
Researchers discovered how to disguise healthy mitochondria so they can slip into diseased cells undetected, extending the lives of mice with a fatal genetic disorder. The breakthrough could one day help children born with rare mitochondrial diseases.
Scientists at Texas Children's Hospital discovered a promising way to treat Rett syndrome, a rare disorder affecting 1 in 10,000 girls. The breakthrough could help 65% of patients by boosting levels of a partially working protein in their brains.
Scientists at Texas Children's Hospital discovered a promising way to treat Rett syndrome, a rare disorder that affects 1 in 10,000 girls and currently has no cure. The breakthrough could help 65% of patients by boosting levels of a partially working protein in their brains.
A groundbreaking gene-editing treatment could reach patients faster than ever before, as Prime Medicine asks the FDA to approve their therapy after just two successful treatments. The drug uses a revolutionary CRISPR technique to fix a genetic disorder that leaves patients vulnerable to deadly infections.
Children with a rare genetic disorder are developing normally thanks to an enzyme therapy crossing the blood-brain barrier. The FDA could approve the treatment this spring, offering hope to families worldwide.
Jeff Allen won $10 million on "Beast Games" and is using his prize to fund research for his son Lucas's rare genetic disorder. His efforts could unlock treatments for millions with Alzheimer's and Parkinson's.
When Julia Friar's newborn son was diagnosed with a life-threatening genetic disorder, she transformed her fear into action. Now she's helping families across the country while fundraising for a cure.
Around 20 people in England with a debilitating genetic condition can now access the first treatment that targets the root cause of their disease. The NHS will provide pegzilarginase to patients with arginase-1 deficiency after breakthrough approval.
Scientists just diagnosed a rare genetic condition in a mother and daughter who lived over 12,000 years ago, proving that communities cared for vulnerable members even during the Ice Age. The discovery marks the earliest genetic diagnosis ever made in human history.
A rare disease nonprofit co-founded by Pearl Jam's Eddie Vedder is revolutionizing medical research by acting like a venture capital firm, turning every dollar into multiple cures. Their innovative funding model has already contributed to three FDA-approved treatments for a devastating skin disease that once had no hope.
A Pennsylvania baby born with a deadly genetic disorder is now thriving after doctors created the world's first personalized CRISPR treatment just for him. Baby KJ can now eat protein and has stopped all medications after three doses of the groundbreaking therapy.
A two-year-old boy in Chennai received a groundbreaking liver transplant from his grandmother, correcting a rare genetic disorder and saving his kidney function. The complex surgery offers hope for other children facing similar life-threatening conditions.
Showing 20 of 25